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2. Clinicopathological definition, management and prognostic value of mogamulizumab‐associated rash and other cutaneous events: A systematic review.

Author

Avallone, G., Roccuzzo, G., Pileri, A., Agostinelli, C., Maronese, C. A., Aquino, C., Tavoletti, G., Onida, F., Fava, P., Ribero, S., Marzano, A. V., Berti, E., Quaglino, P., and Alberti‐Violetti, S.

Subjects
PROGNOSIS, SEZARY syndrome, PHOTOSENSITIVITY disorders, DIAGNOSIS, MONOCLONAL antibodies
Abstract

Mogamulizumab is a first‐in‐class IgG1k monoclonal antibody that selectively targets the chemokine receptor type 4. The drug has received Food and Drug administration authorisation for mycosis fungoides and Sézary syndrome following failure of at least one previous course of systemic therapy and now is available in Europe. One of the most common treatment‐related side effects observed has been the mogamulizumab‐associated rash (MAR), which affects up to a quarter of patients and is the most frequent adverse event leading to drug discontinuation. The aim of this study is to perform a systematic review of the literature on patients diagnosed with MAR and other mogamulizumab‐related cutaneous events to describe the clinical and histological characteristics, the management in clinical practice and to assess whether these events have prognostic implications. In total, 2073 records were initially identified through a literature search, 843 of which were duplicates. After screening for eligibility and inclusion criteria, 49 articles reporting mogamulizumab‐associated cutaneous events were included. Totally, 1516 patients were retrieved, with a slight male prevalence as for the available data (639 males and 570 females, i.e. 52.9% vs. 47.1%). Regarding the reported clinicopathological findings of the cutaneous reactions, the five most common patterns were spongiotic/psoriasiform dermatitis (22%), eruptions characterized by the presence of papules and/or plaques (16.1%), cutaneous granulomatosis (11.4%), morbilliform or erythrodermic dermatitis (9.4%) and photodermatitis (7.1%). Our results highlight how the majority of the reported cutaneous adverse events on mogamulizumab are of mild‐to‐moderate entity and generally manageable in clinical practice, though prompt recognition is essential and case‐by‐case assessment should be recommended. Future research will need to focus on the MAR prognostic implications and to identify genomic and molecular markers for a more rapid and accurate diagnosis. [ABSTRACT FROM AUTHOR]

Published
2024
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3. Allogeneic hematopoietic stem cell transplantation for advanced mycosis fungoides and Sézary syndrome. An updated experience of the Lymphoma Working Party of the European Society for Blood and Marrow Transplantation

Author

Domingo-Domenech, E., Duarte, R. F., Boumedil, A., Onida, F., Gabriel, I., Finel, H., Arcese, W., Browne, P., Beelen, D., Kobbe, G., Veelken, H., Arranz, R., Greinix, H., Lenhoff, S., Poiré, X., Ribera, J. M., Thompson, J., Zuckerman, T., Mufti, G. J., Cortelezzi, A., Olavarria, E., Dreger, P., Sureda, A., and Montoto, S.

Published
2021
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4. Communicating the diagnosis of a hematological neoplastic disease to patients’ minor children: a multicenter prospective study

Author

Manghisi, B, Borin, L, Monaco, M, Sacco, G, Antolini, L, Mantegazza, R, Barichello, M, Mazza, U, Zappasodi, P, Onida, F, Arcaini, L, Cairoli, R, Gambacorti Passerini, C, Manghisi, Beatrice, Borin, Lorenza, Monaco, Maria Rosaria, Sacco, Gaia Giulia Angela, Antolini, Laura, Mantegazza, Raffaele, Barichello, Monica, Mazza, Umberto, Zappasodi, Patrizia, Onida, Francesco, Arcaini, Luca, Cairoli, Roberto, Gambacorti Passerini, Carlo, Manghisi, B, Borin, L, Monaco, M, Sacco, G, Antolini, L, Mantegazza, R, Barichello, M, Mazza, U, Zappasodi, P, Onida, F, Arcaini, L, Cairoli, R, Gambacorti Passerini, C, Manghisi, Beatrice, Borin, Lorenza, Monaco, Maria Rosaria, Sacco, Gaia Giulia Angela, Antolini, Laura, Mantegazza, Raffaele, Barichello, Monica, Mazza, Umberto, Zappasodi, Patrizia, Onida, Francesco, Arcaini, Luca, Cairoli, Roberto, and Gambacorti Passerini, Carlo

Abstract

Background When a hematological malignancy is diagnosed, the whole family carries the burden of the disease; parents often try to protect minor children from suffering by avoiding communication about their disease. Since 2009, patients with minors at the Adult Hematology Division at San Gerardo Hospital (Monza) can take part in the "Emanuela Project": children can visit parents and talk with psychologists and hematologists, who explain the disease through simple metaphors.Materials and Methods The EMY STUDY aimed to evaluate the impact of illness-related communication on children's behavior, comparing Monza's experience with other Hematology Units, where the communication is delegated to parents or psychological support. Questionnaires exploring the children's main behaviors (school performance, appetite, sleeping patterns, attachment to family figures, and family dialogue) were administered to both sick (SP) and healthy (HP) parents. From 2017 to 2021, 32 patients were enrolled, 20 from Monza and 12 from other hospitals; 84 questionnaires were globally collected.Results In Monza's group, no major changes in children's behavior were observed and an open dialogue about the disease was often possible. Disease communication is considered crucial and perceived as a responsibility of parents together with a professional figure, mainly the hematologist. Patients were satisfied with "Emanuela Project," reporting positive effects on doctor-patient relationship. Difficulties in separation were significantly higher at other hospitals (P = .019) than in Monza. While at other centers communication is considered parents' responsibility, Monza's patients emphasize the role of professional figures (P = .007). Differently from other hospitals, the role of the hematologist is crucial to Monza's patients (P = .001).Conclusion Disease communication to patients' offspring is a crucial moment in the process of care, and the hematologist can play a major role in this difficult task, with

Published
2024

5. Outcomes of CMML patients undergoing allo-HCT are significantly worse compared to MDS-a study of the CMWP of the EBMT

Author

Rovó, A, Gras, L, Piepenbroek, B, Kroeger, N, Reinhardt, HC, Radujkovic, A, Blaise, D, Kobbe, G, Niityvuopio, R, Platzbecker, U, Sockel, K, Hunault-Berger, M, Cornelissen, JJ, Forcade, E, Bourhis, JH, Chalandon, Y, Kinsella, F, Nguyen-Quoc, S, Maertens, J, Elmaagacli, A, Mordini, N, Hayden, P, Raj, K, Drozd-Sokolowska, J, de Wreede, LC, Mclornan, DP, Robin, M, Yakoub-Agha, I, Onida, F, Rovó, A, Gras, L, Piepenbroek, B, Kroeger, N, Reinhardt, HC, Radujkovic, A, Blaise, D, Kobbe, G, Niityvuopio, R, Platzbecker, U, Sockel, K, Hunault-Berger, M, Cornelissen, JJ, Forcade, E, Bourhis, JH, Chalandon, Y, Kinsella, F, Nguyen-Quoc, S, Maertens, J, Elmaagacli, A, Mordini, N, Hayden, P, Raj, K, Drozd-Sokolowska, J, de Wreede, LC, Mclornan, DP, Robin, M, Yakoub-Agha, I, and Onida, F

Abstract

Although CMML since long has been separated from MDS, many studies continue to evaluate the outcomes of both diseases after hematopoietic cell transplantation (allo-HCT) together. Data evaluating outcomes of a large CMML cohort after allo-HCT compared to MDS are limited. We aim to compare outcomes of CMML to MDS patients who underwent allo-HCT between 2010 and 2018. Patients ≥18 years with CMML and MDS undergoing allo-HCT reported to the EBMT registry were analyzed. Progression to AML before allo-HCT was an exclusion criterion. Overall survival (OS), progression/relapse-free survival (PFS), relapse incidence (including progression) (REL), and non-relapse mortality (NRM) were evaluated in univariable and multivariable (MVA) Cox proportional hazard models including interaction terms between disease and confounders. In total, 10832 patients who underwent allo-HCT were included in the study, there were a total of 1466 CMML, and 9366 MDS. The median age at time of allo-HCT in CMML (median 60.5, IQR 54.3–65.2 years) was significantly higher than in the MDS cohort (median 58.8, IQR 50.2–64.5 years; p < .001). A significantly higher percentage of CMML patients were male (69.4%) compared to MDS (61.2%; p < .001). There were no clinically meaningful differences in the distribution of Karnofsky score, Sorror HCT-CI score at allo-HCT, and donor type, between the CMML and MDS patients. RIC platforms were utilized in 63.9% of CMML allo-HCT, and in 61.4% of MDS patients (p = .08). In univariable analyses, we found that OS, PFS, and REL were significantly worse in CMML when compared with MDS (all p < .0001), whereas no significant difference was observed in NRM (p = .77). In multivariable analyses, the HR comparing MDS versus CMML for OS was 0.81 (95% CI, 0.74–0.88, p < .001), PFS 0.76 (95% CI 0.70–0.82, p < .001), relapse 0.66 (95% CI 0.59–0.74, p < .001), and NRM 0.87 (95% CI 0.78–0.98, p = .02), respectively. The association between baseline variables and outco

Published
2024

6. Impact of post-transplant cyclophosphamide (PTCy)-based prophylaxis in matched sibling donor allogeneic haematopoietic cell transplantation for patients with myelodysplastic syndrome: a retrospective study on behalf of the Chronic Malignancies Working Party of the EBMT

Author

Salas, M. Q., Eikema, D. -J., Koster, L., Maertens, J., Passweg, J., Finke, J., Broers, A. E. C., Koc, Y., Kroger, N., Ozkurt, Z. N., Pascual-Cascon, M. J., Platzbecker, U., Van Gorkom, G., Schroeder, T., Lopez-Lorenzo, J. L., Martino, Michelangelo, Chiusolo, Patrizia, Kaufmann, M., Onida, F., Gurnari, C., Scheid, C., Drozd-Sokolowska, J., Raj, K., Robin, M., Mclornan, D. P., Martino M., Chiusolo P. (ORCID:0000-0002-1355-1587), Salas, M. Q., Eikema, D. -J., Koster, L., Maertens, J., Passweg, J., Finke, J., Broers, A. E. C., Koc, Y., Kroger, N., Ozkurt, Z. N., Pascual-Cascon, M. J., Platzbecker, U., Van Gorkom, G., Schroeder, T., Lopez-Lorenzo, J. L., Martino, Michelangelo, Chiusolo, Patrizia, Kaufmann, M., Onida, F., Gurnari, C., Scheid, C., Drozd-Sokolowska, J., Raj, K., Robin, M., Mclornan, D. P., Martino M., and Chiusolo P. (ORCID:0000-0002-1355-1587)

Abstract

We retrospectively compared outcomes of 404 MDS patients undergoing 1st matched sibling donor allo-HCT receiving either PTCy-based (n = 66) or other “conventional prophylaxis” (n = 338; mostly calcineurin inhibitor + methotrexate or MMF). Baseline characteristics were balanced, except for higher use of myeloablative regimens in the PTCy group (52.3% vs. 38.2%, p = 0.047). Incidences of neutrophil (Day +28: 89% vs. 97%, p = 0.011) and platelet (Day +100: 89% vs. 97%, p < 0.001) engraftment were lower for PTCy-based. Day +100 cumulative incidences of grade II–IV and III–IV aGVHD, and 5-year CI of extensive cGVHD were 32%, 18% and 18% for PTCy-based and 25% (p = 0.3), 13% (p = 0.4) and 31% (p = 0.09) for the conventional cohort. Five-year OS (51% vs. 52%, p = 0.6) and GRFS (33% vs. 25%, p = 0.6) were similar between groups. Patients receiving PTCy had a trend to a lower cumulative incidence of relapse (20% vs. 33%, p = 0.06), not confirmed on multivariable analysis (p = 0.3). Although higher NRM rates were observed in patients receiving PTCy (32% vs. 21%, p = 0.02) on univariate analysis, this was not confirmed on multivariate analysis (HR 1.46, p = 0.18), and there was no resultant effect on OS (HR 1.20, p = 0.5). Based on these data, PTCy prophylaxis appears to be an attractive option for patients with MDS undergoing MSD allo-HCT.

Published
2024

7. Nilotinib in steroid-refractory cGVHD: prospective parallel evaluation of response, according to NIH criteria and exploratory response criteria (GITMO criteria)

Author

Olivieri, A., Mancini, G., Olivieri, J., Marinelli Busilacchi, E., Cimminiello, M., Pascale, S. P., Nuccorini, R., Patriarca, F., Corradini, P., Bacigalupo, A., Angelini, S., Poloni, A., Grillo, G., Onida, F., Martino, M., Di Renzo, N., Nagler, A., Mordini, N., Bruno, B., Ciceri, F., and Bonifazi, F.

Published
2020
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8. Global patterns of care in advanced stage mycosis fungoides/Sezary syndrome: a multicenter retrospective follow-up study from the Cutaneous Lymphoma International Consortium

Author

Quaglino, P., Maule, M., Prince, H.M., Porcu, P., Horwitz, S., Duvic, M., Talpur, R., Vermeer, M., Bagot, M., Guitart, J., Papadavid, E., Sanches, J.A., Hodak, E., Sugaya, M., Berti, E., Ortiz-Romero, P., Pimpinelli, N., Servitje, O., Pileri, A., Zinzani, P.L., Estrach, T., Knobler, R., Stadler, R., Fierro, M.T., Alberti Violetti, S., Amitay-Laish, I., Antoniou, C., Astrua, C., Chaganti, S., Child, F., Combalia, A., Fabbro, S., Fava, P., Grandi, V., Jonak, C., Martinez-Escala, E., Kheterpal, M., Kim, E.J., McCormack, C., Miyagaki, T., Miyashiro, D., Morris, S., Muniesa, C., Nikolaou, V., Ognibene, G., Onida, F., Osella-Abate, S., Porkert, S., Postigo-Llorente, C., Ram-Wolff, C., Ribero, S., Rogers, K., Sanlorenzo, M., Stranzenbach, R., Spaccarelli, N., Stevens, A., Zugna, D., Rook, A.H., Geskin, L.J., Willemze, R., Whittaker, S., Hoppe, R., Scarisbrick, J., and Kim, Y.

Published
2017
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9. Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller F. R., Snowden J. A., Corbacioglu S., Greco R., Alexander T., Snowden J., Badoglio M., Labopin M., Abinun M., Apte S., Arnold R., Domenech A., Brierley C., Burman J., Castilla-Llorente C., Cooper N., Daghia G., Daikeler T., del Papa N., de Vries-Bouwstra J., Farge D., Finke J., Hagglund H., Hawkey C., Henes J., Hiepe F., Jessop H., Kiely D., Kazmi M., Kirgizov K., Kramer E., Mancardi G., Marjanovic Z., Martin R., Martin T., Ma D., Moore J., Miller P., Muraro P., Oliveira M. -C., Polushin A., Onida F., Simoes B., Puyade M., Resnick I., Ricart E., Rovira M., Saccardi R., Saif M., Sakellari I., Sharrack B., Snarski E., Scherer H. U., Sossa C., Withers B., Wulffraat N., Zaccara E., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Diaz C. H., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Vincent M. G., Handgretinger R., Kleinschmidt K., Lawitschka A., Martinez A. P., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., Yesilipek A., Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller F. R., Snowden J. A., Corbacioglu S., Greco R., Alexander T., Snowden J., Badoglio M., Labopin M., Abinun M., Apte S., Arnold R., Domenech A., Brierley C., Burman J., Castilla-Llorente C., Cooper N., Daghia G., Daikeler T., del Papa N., de Vries-Bouwstra J., Farge D., Finke J., Hagglund H., Hawkey C., Henes J., Hiepe F., Jessop H., Kiely D., Kazmi M., Kirgizov K., Kramer E., Mancardi G., Marjanovic Z., Martin R., Martin T., Ma D., Moore J., Miller P., Muraro P., Oliveira M. -C., Polushin A., Onida F., Simoes B., Puyade M., Resnick I., Ricart E., Rovira M., Saccardi R., Saif M., Sakellari I., Sharrack B., Snarski E., Scherer H. U., Sossa C., Withers B., Wulffraat N., Zaccara E., Amrolia P., Ansari M., Balduzzi A., Dalassier A., Dalle J. -H., Diaz C. H., Feuchtinger T., Locatelli F., Lucchini G., Galimard J. -E., Vincent M. G., Handgretinger R., Kleinschmidt K., Lawitschka A., Martinez A. P., Peters C., Rocha V., Ruggeri A., Sedlacek P., Svec P., Toporski J., and Yesilipek A.

Abstract

Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long-term disease control or cure, but therapy-related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence-based interspeciality guidelines and recommendations.

Published
2022

10. ‘Real-life’ report on the management of chronic GvHD in the Gruppo Italiano Trapianto Midollo Osseo (GITMO)

Author

Giaccone, L, Mancini, G, Mordini, N, Gargiulo, G, De Cecco, V, Angelini, S, Arpinati, M, Baronciani, D, Bozzoli, V, Bramanti, S, Calore, E, Cavattoni, I M, Cimminiello, M, Colombo, A A, Facchini, L, Falcioni, S, Faraci, M, Fedele, R, Guidi, S, Iori, A P, Marotta, S, Micò, M C, Milone, G, Onida, F, Pastore, D, Patriarca, F, Pini, M, Raimondi, R, Rovelli, A, Santarone, S, Severino, A, Skert, C, Stanghellini, M T L, Tecchio, C, Vassallo, E, Chiarucci, M, Bruno, B, Bonifazi, F, and Olivieri, A

Published
2018
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11. POS1314 AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION: LONG-TERM OUTCOMES IN A SINGLE-CENTER COHORT OF PATIENTS WITH SYSTEMIC SCLEROSIS

Author

Del Papa, N., primary, Rindone, A., additional, Cavalli, S., additional, Minniti, A., additional, Onida, F., additional, Saporiti, G., additional, Goldaniga, M., additional, Iannone, C., additional, Armentaro, G., additional, Germinario, S., additional, Sette, M., additional, and Caporali, R., additional

Published
2023
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12. Practice harmonization workshops of EBMT: an expert-based approach to generate practical and contemporary guidelines within the arena of hematopoietic cell transplantation and cellular therapy.

Author

Yakoub-Agha, I., Greco, R., Onida, F., Cámara, R. de la, Ciceri, F., Corbacioglu, S., Dolstra, H., Glass, B., Kenyon, M., McLornan, D.P., Neven, B., Latour, R.P. de, Peric, Z., Ruggeri, A., Snowden, J.A., Sureda, A., Sánchez-Ortega, I., Yakoub-Agha, I., Greco, R., Onida, F., Cámara, R. de la, Ciceri, F., Corbacioglu, S., Dolstra, H., Glass, B., Kenyon, M., McLornan, D.P., Neven, B., Latour, R.P. de, Peric, Z., Ruggeri, A., Snowden, J.A., Sureda, A., and Sánchez-Ortega, I.

Abstract

01 juni 2023, Item does not contain fulltext, For hematopoietic cell transplantation (HCT) and cellular therapy (CT), clinical patient care is localized, and practices may differ between countries and from center to center even within the same country. Historically, international guidelines were not always adapted to the changing daily clinical practice and practical topics there were not always addressed. In the absence of well-established guidelines, centers tended to develop local procedures/policies, frequently with limited communication with other centers. To try to harmonize localized clinical practices for malignant and non-malignant hematological disorders within EBMT scope, the practice harmonization and guidelines (PH&G) committee of the EBMT will co-ordinate workshops with topic-specific experts from interested centers. Each workshop will discuss a specific issue and write guidelines/recommendations that practically addresses the topic under review. To provide clear, practical and user-friendly guidelines when international consensus is lacking, the EBMT PH&G committee plans to develop European guidelines by HCT and CT physicians for peers' use. Here, we define how workshops will be conducted and guidelines/recommendations produced, approved and published. Ultimately, there is an aspiration for some topics, where there is sufficient evidence base to be considered for systematic reviews, which are a more robust and future-proofed basis for guidelines/recommendations than consensus opinion.

Published
2023

13. Immune effector cell–associated hematotoxicity: EHA/EBMT consensus grading and best practice recommendations

Author

Rejeski, K, Subklewe, M, Aljurf, M, Bachy, E, Balduzzi, A, Barba, P, Bruno, B, Benjamin, R, Carrabba, M, Chabannon, C, Ciceri, F, Corradini, P, Delgado, J, Di Blasi, R, Greco, R, Houot, R, Iacoboni, G, Jaeger, U, Kersten, M, Mielke, S, Nagler, A, Onida, F, Peric, Z, Roddie, C, Ruggeri, A, Sanchez-Guijo, F, Sánchez-Ortega, I, Schneidawind, D, Schubert, M, Snowden, J, Thieblemont, C, Topp, M, Zinzani, P, Gribben, J, Bonini, C, Sureda Balari, A, Yakoub-Agha, I, Rejeski, Kai, Subklewe, Marion, Aljurf, Mahmoud, Bachy, Emmanuel, Balduzzi, Adriana Cristina, Barba, Pere, Bruno, Benedetto, Benjamin, Reuben, Carrabba, Matteo Giovanni, Chabannon, Christian, Ciceri, Fabio, Corradini, Paolo, Delgado, Julio, Di Blasi, Roberta, Greco, Raffaella, Houot, Roch, Iacoboni, Gloria, Jaeger, Ulrich, Kersten, Marie José, Mielke, Stephan, Nagler, Arnon, Onida, Francesco, Peric, Zinaida, Roddie, Claire, Ruggeri, Annalisa, Sanchez-Guijo, Fermin M, Sánchez-Ortega, Isabel, Schneidawind, Dominik, Schubert, Maria-Luisa, Snowden, John, Thieblemont, Catherine, Topp, Max S, Zinzani, Pierluigi Luigi, Gribben, John G, Bonini, Chiara, Sureda Balari, Anna, Yakoub-Agha, Ibrahim, Rejeski, K, Subklewe, M, Aljurf, M, Bachy, E, Balduzzi, A, Barba, P, Bruno, B, Benjamin, R, Carrabba, M, Chabannon, C, Ciceri, F, Corradini, P, Delgado, J, Di Blasi, R, Greco, R, Houot, R, Iacoboni, G, Jaeger, U, Kersten, M, Mielke, S, Nagler, A, Onida, F, Peric, Z, Roddie, C, Ruggeri, A, Sanchez-Guijo, F, Sánchez-Ortega, I, Schneidawind, D, Schubert, M, Snowden, J, Thieblemont, C, Topp, M, Zinzani, P, Gribben, J, Bonini, C, Sureda Balari, A, Yakoub-Agha, I, Rejeski, Kai, Subklewe, Marion, Aljurf, Mahmoud, Bachy, Emmanuel, Balduzzi, Adriana Cristina, Barba, Pere, Bruno, Benedetto, Benjamin, Reuben, Carrabba, Matteo Giovanni, Chabannon, Christian, Ciceri, Fabio, Corradini, Paolo, Delgado, Julio, Di Blasi, Roberta, Greco, Raffaella, Houot, Roch, Iacoboni, Gloria, Jaeger, Ulrich, Kersten, Marie José, Mielke, Stephan, Nagler, Arnon, Onida, Francesco, Peric, Zinaida, Roddie, Claire, Ruggeri, Annalisa, Sanchez-Guijo, Fermin M, Sánchez-Ortega, Isabel, Schneidawind, Dominik, Schubert, Maria-Luisa, Snowden, John, Thieblemont, Catherine, Topp, Max S, Zinzani, Pierluigi Luigi, Gribben, John G, Bonini, Chiara, Sureda Balari, Anna, and Yakoub-Agha, Ibrahim

Abstract

Hematological toxicity is the most common adverse event after chimeric antigen receptor (CAR) T-cell therapy. Cytopenias can be profound and long-lasting and can predispose for severe infectious complications. In a recent worldwide survey, we demonstrated that there remains considerable heterogeneity in regard to current practice patterns. Here, we sought to build consensus on the grading and management of immune effector cell–associated hematotoxicity (ICAHT) after CAR T-cell therapy. For this purpose, a joint effort between the European Society for Blood and Marrow Transplantation (EBMT) and the European Hematology Association (EHA) involved an international panel of 36 CAR T-cell experts who met in a series of virtual conferences, culminating in a 2-day meeting in Lille, France. On the basis of these deliberations, best practice recommendations were developed. For the grading of ICAHT, a classification system based on depth and duration of neutropenia was developed for early (day 0-30) and late (after day +30) cytopenia. Detailed recommendations on risk factors, available preinfusion scoring systems (eg, CAR-HEMATOTOX score), and diagnostic workup are provided. A further section focuses on identifying hemophagocytosis in the context of severe hematotoxicity. Finally, we review current evidence and provide consensus recommendations for the management of ICAHT, including growth factor support, anti-infectious prophylaxis, transfusions, autologous hematopoietic stem cell boost, and allogeneic hematopoietic cell transplantation. In conclusion, we propose ICAHT as a novel toxicity category after immune effector cell therapy, provide a framework for its grading, review literature on risk factors, and outline expert recommendations for the diagnostic workup and short- and long-term management.

Published
2023

14. Busulfan or Treosulfan Conditioning Platform for Allogeneic Stem Cell Transplantation in Patients Aged >60 y with Acute Myeloid Leukemia/Myelodysplastic Syndrome: A Subanalysis of the GITMO AlloEld Study

Author

Malagola, M., Polverelli, N., Martino, Michelangelo, Patriarca, Fabrizio, Bruno, Brunella, Giaccone, L., Grillo, G., Bramanti, S., Bernasconi, P., De Gobbi, M., Natale, A., Terruzzi, E., Olivieri, Alessandra, Chiusolo, Patrizia, Carella, A. M., Casini, Marina, Maffini, E., Nozzoli, C., Mazza, P., Bassi, S., Onida, F., Vacca, Alessandro, Falcioni, S., Luppi, M., Iori, A. P., Pavone, V., Skert, C., Carluccio, P., Borghero, C., Proia, A., Selleri, C., Rubini, V., Sacchi, N., Oldani, E., Bonifazi, F., Ciceri, F., Russo, Daniele, Bernardi, S., Farina, M., Fiore, M., Lupo Stanghellini, M. T., Fanin, R., Faraci, D. G., Castagna, Luigi, Colombo, A. A., Nicoli, P., Santarone, S., Scortechini, I., Metafuni, Elisabetta, Merla, E., Cavattoni, I., Cutini, I., Mazzone, A., Saporiti, G., Canale, F. A., Piras, Edoardo, Galieni, P., Debbia, G., La Rocca, U., Mele, Dario Antonio, Carobolante, F., Elice, F., Fanelli, F., Martino M., Patriarca F., Bruno B., Olivieri A., Chiusolo P. (ORCID:0000-0002-1355-1587), Casini M. (ORCID:0000-0002-3209-7770), Vacca A., Russo D., Castagna L., Metafuni E., Piras E., Mele A., Malagola, M., Polverelli, N., Martino, Michelangelo, Patriarca, Fabrizio, Bruno, Brunella, Giaccone, L., Grillo, G., Bramanti, S., Bernasconi, P., De Gobbi, M., Natale, A., Terruzzi, E., Olivieri, Alessandra, Chiusolo, Patrizia, Carella, A. M., Casini, Marina, Maffini, E., Nozzoli, C., Mazza, P., Bassi, S., Onida, F., Vacca, Alessandro, Falcioni, S., Luppi, M., Iori, A. P., Pavone, V., Skert, C., Carluccio, P., Borghero, C., Proia, A., Selleri, C., Rubini, V., Sacchi, N., Oldani, E., Bonifazi, F., Ciceri, F., Russo, Daniele, Bernardi, S., Farina, M., Fiore, M., Lupo Stanghellini, M. T., Fanin, R., Faraci, D. G., Castagna, Luigi, Colombo, A. A., Nicoli, P., Santarone, S., Scortechini, I., Metafuni, Elisabetta, Merla, E., Cavattoni, I., Cutini, I., Mazzone, A., Saporiti, G., Canale, F. A., Piras, Edoardo, Galieni, P., Debbia, G., La Rocca, U., Mele, Dario Antonio, Carobolante, F., Elice, F., Fanelli, F., Martino M., Patriarca F., Bruno B., Olivieri A., Chiusolo P. (ORCID:0000-0002-1355-1587), Casini M. (ORCID:0000-0002-3209-7770), Vacca A., Russo D., Castagna L., Metafuni E., Piras E., and Mele A.

Abstract

Background. The conditioning regimens with different alkylators at different doses can influence the outcome of allogeneic stem cell transplantation (SCT), but conclusive data are missing. Methods. With the aim to analyze real-life allogeneic SCTs performed in Italy between 2006 and 2017 in elderly patients (aged >60 y) with acute myeloid leukemia or myelodysplastic syndrome, we collected 780 first transplants data. For analysis purposes, patients were grouped according to the type of alkylator included in the conditioning (busulfan [BU]-based; n = 618; 79%; treosulfan [TREO]-based; n=162; 21%). Results. No significant differences were observed in nonrelapse mortality, cumulative incidence of relapse, and overall survival, although in the TREO-based group, we observed a greater proportion of elderly patients (P < 0.001); more active diseases at the time of SCT (P < 0.001); a higher prevalence of patients with either hematopoietic cell transplantation-comorbidity index ≥3 (P < 0.001) or a good Karnofsky performance status (P = 0.025); increased use of peripheral blood stem cells as graft sources (P < 0.001); and greater use of reduced intensity conditioning regimens (P = 0.013) and of haploidentical donors (P < 0.001). Moreover, the 2-y cumulative incidence of relapse with myeloablative doses of BU was significantly lower than that registered with reduced intensity conditioning (21% versus 31%; P = 0.0003). This was not observed in the TREO-based group. Conclusions. Despite a higher number of risk factors in the TREO group, no significant differences were observed in nonrelapse mortality, cumulative incidence of relapse, and overall survival according to the type of alkylator, suggesting that TREO has no advantage over BU in terms of efficacy and toxicity in acute myeloid leukemia and myelodysplastic syndrome.

Published
2023

15. Haematopoietic stem cell transplantation for severe autoimmune diseases in children : a review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini‐Gutzwiller, FR, Snowden, JA, Corbacioglu, S, Greco, R, Alexander, T, Snowden, J, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla‐Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries‐Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, HU, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, CH, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, MG, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, AP, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, Yesilipek, A, Achini-Gutzwiller, F, Snowden, J, Corbacioglu, S, Greco, R, Alexander, T, Badoglio, M, Labopin, M, Abinun, M, Apte, S, Arnold, R, Domenech, A, Brierley, C, Burman, J, Castilla-Llorente, C, Cooper, N, Daghia, G, Daikeler, T, del Papa, N, de Vries-Bouwstra, J, Farge, D, Finke, J, Hagglund, H, Hawkey, C, Henes, J, Hiepe, F, Jessop, H, Kiely, D, Kazmi, M, Kirgizov, K, Kramer, E, Mancardi, G, Marjanovic, Z, Martin, R, Martin, T, Ma, D, Moore, J, Miller, P, Muraro, P, Oliveira, M, Polushin, A, Onida, F, Simoes, B, Puyade, M, Resnick, I, Ricart, E, Rovira, M, Saccardi, R, Saif, M, Sakellari, I, Sharrack, B, Snarski, E, Scherer, H, Sossa, C, Withers, B, Wulffraat, N, Zaccara, E, Amrolia, P, Ansari, M, Balduzzi, A, Dalassier, A, Dalle, J, Diaz, C, Feuchtinger, T, Locatelli, F, Lucchini, G, Galimard, J, Vincent, M, Handgretinger, R, Kleinschmidt, K, Lawitschka, A, Martinez, A, Peters, C, Rocha, V, Ruggeri, A, Sedlacek, P, Svec, P, Toporski, J, and Yesilipek, A

Subjects
autoimmune diseases, haematopoietic stem cell transplantation, paediatric, ddc:610, paediatric, surgical procedures, operative, Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA, haematopoietic stem cell transplantation, 610 Medizin, autoimmune diseases, autoimmune disease, Hematology
Abstract

Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (

Published
2022

16. Decision analysis of allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome stratified according to the revised International Prognostic Scoring System

Author

Della Porta, M G, Jackson, C H, Alessandrino, E P, Rossi, M, Bacigalupo, A, van Lint, M T, Bernardi, M, Allione, B, Bosi, A, Guidi, S, Santini, V, Malcovati, L, Ubezio, M, Milanesi, C, Todisco, E, Voso, M T, Musto, P, Onida, F, Iori, A P, Cerretti, R, Grillo, G, Molteni, A, Pioltelli, P, Borin, L, Angelucci, E, Oldani, E, Sica, S, Pascutto, C, Ferretti, V, Santoro, A, Bonifazi, F, Cazzola, M, and Rambaldi, A

Published
2017
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19. Changes in Stem Cell Transplant activity and procedures during SARS-CoV2 pandemic in Italy: an Italian Bone Marrow Transplant Group (GITMO) nationwide analysis (TransCOVID-19 Survey)

Author

Russo, D, Polverelli, N, Malagola, M, Farina, M, Leoni, A, Bernardi, S, Mammoliti, S, Sacchi, N, Martino, M, Ciceri, F, Zallio, F, Olivieri, A, Falcioni, S, Storti, G, Michieli, M, Carluccio, P, Grassi, A, Oldani, E, Bonifazi, F, Prete, A, Cavattoni, I, Maffeis, M, Pastore, D, Vacca, A, Caravelli, D, Mirabile, M, Mordini, N, Nozzoli, C, Faraci, M, Federico, V, Ronconi, S, Skert, C, Onida, F, Marcatti, M, Piemontese, S, Narni, F, Balduzzi, A, De Simone, G, Picardi, A, De Gobbi, M, Calore, E, Tringali, S, Zecca, M, Secondino, S, Guiducci, B, Pelosini, M, Zuffa, E, Facchini, L, Imola, M, Iori, A, Proia, A, Sica, S, Armiento, D, Carella, A, Dellacasa, C, Fagioli, F, Rabusin, M, Ferrario, A, Elice, F, Russo D., Polverelli N., Malagola M., Farina M., Leoni A., Bernardi S., Mammoliti S., Sacchi N., Martino M., Ciceri F., Zallio F., Olivieri A., Falcioni S., Storti G., Michieli M., Carluccio P., Grassi A., Oldani E., Bonifazi F., Prete A., Cavattoni I. M., Maffeis M., Pastore D., Vacca A., Caravelli D., Mirabile M., Mordini N., Nozzoli C., Faraci M., Federico V., Ronconi S., Skert C., Onida F., Marcatti M., Piemontese S., Narni F., Balduzzi A., De Simone G., Picardi A., De Gobbi M., Calore E., Tringali S., Zecca M., Secondino S., Guiducci B., Pelosini M., Zuffa E., Facchini L., Imola M., Iori A. P., Proia A., Sica S., Armiento D., Carella A. M., Dellacasa C. M., Fagioli F., Rabusin M., Ferrario A., Elice F., Russo, D, Polverelli, N, Malagola, M, Farina, M, Leoni, A, Bernardi, S, Mammoliti, S, Sacchi, N, Martino, M, Ciceri, F, Zallio, F, Olivieri, A, Falcioni, S, Storti, G, Michieli, M, Carluccio, P, Grassi, A, Oldani, E, Bonifazi, F, Prete, A, Cavattoni, I, Maffeis, M, Pastore, D, Vacca, A, Caravelli, D, Mirabile, M, Mordini, N, Nozzoli, C, Faraci, M, Federico, V, Ronconi, S, Skert, C, Onida, F, Marcatti, M, Piemontese, S, Narni, F, Balduzzi, A, De Simone, G, Picardi, A, De Gobbi, M, Calore, E, Tringali, S, Zecca, M, Secondino, S, Guiducci, B, Pelosini, M, Zuffa, E, Facchini, L, Imola, M, Iori, A, Proia, A, Sica, S, Armiento, D, Carella, A, Dellacasa, C, Fagioli, F, Rabusin, M, Ferrario, A, Elice, F, Russo D., Polverelli N., Malagola M., Farina M., Leoni A., Bernardi S., Mammoliti S., Sacchi N., Martino M., Ciceri F., Zallio F., Olivieri A., Falcioni S., Storti G., Michieli M., Carluccio P., Grassi A., Oldani E., Bonifazi F., Prete A., Cavattoni I. M., Maffeis M., Pastore D., Vacca A., Caravelli D., Mirabile M., Mordini N., Nozzoli C., Faraci M., Federico V., Ronconi S., Skert C., Onida F., Marcatti M., Piemontese S., Narni F., Balduzzi A., De Simone G., Picardi A., De Gobbi M., Calore E., Tringali S., Zecca M., Secondino S., Guiducci B., Pelosini M., Zuffa E., Facchini L., Imola M., Iori A. P., Proia A., Sica S., Armiento D., Carella A. M., Dellacasa C. M., Fagioli F., Rabusin M., Ferrario A., and Elice F.

Abstract

The Transplant Centers belonging to Gruppo Italiano per il Trapianto di Midollo Osseo (GITMO) conducted a survey with the aim of evaluating the effect of SARS-CoV2 pandemic on the allogeneic transplant activity in Italy. The pandemic period from 1/3/2020 to 31/7/2020 was compared with the same period in 2019. Overall, in 2020 there was a 2.4% reduction in the number of allo-HCT cases compared to 2019. Interestingly, this deflection did not affect the acute leukemia cases (+5.7% in 2020). The use of peripheral blood-derived stem cells (+10.7%) and cryopreservation (97.4% of the centers) was highly adopted in 2020. Despite the sanitary emergency, almost all of the surveyed centers declared no impact of SARS-CoV2 pandemic on the transplant timing and outcomes, and the sanitary policy was positively evaluated by the majority of centers. The emergency measures ensured that only a minority of the allo-HCT patients had been infected by SARS-CoV2; however, a mortality of 42.1% among the allo-HCT patients hospitalized for COVID-19 was recorded. This survey gives us the information that the GITMO Group reacted positively to the pandemic. Thanks to the emergency strategies, the Italian allo-HCT activity continued safely, showing only a minor deflection and offering the same probability of cure to the transplanted patients.

Published
2021

21. OP021 Autologous haematopoietic stem cell transplantation for Crohn’s disease: a retrospective study from the European Society for Blood & Marrow Transplantation (EBMT) Autoimmune Diseases Working Party

Author

Brierley, C, Castilla-Llorente, C, Labopin, M, Badoglio, M, Rovira, M, Ricart, E, Dierickx, D, Vermeire, S, Hasselblatt, P, Finke, J, Onida, F, Cassinotti, A, Satsangi, J, Kazmi, M, López-Sanromán, A, Farge, D, Travis, S, Hawkey, C, and Snowden, J

Published
2018
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22. Changes in Stem Cell Transplant activity and procedures during SARS-CoV2 pandemic in Italy: an Italian Bone Marrow Transplant Group (GITMO) nationwide analysis (TransCOVID-19 Survey)

Author

Russo D., Polverelli N., Malagola M., Farina M., Leoni A., Bernardi S., Mammoliti S., Sacchi N., Martino M., Ciceri F., Zallio F., Olivieri A., Falcioni S., Storti G., Michieli M., Carluccio P., Grassi A., Oldani E., Bonifazi F., Prete A., Cavattoni I. M., Maffeis M., Pastore D., Vacca A., Caravelli D., Mirabile M., Mordini N., Nozzoli C., Faraci M., Federico V., Ronconi S., Skert C., Onida F., Marcatti M., Piemontese S., Narni F., Balduzzi A., De Simone G., Picardi A., De Gobbi M., Calore E., Tringali S., Zecca M., Secondino S., Guiducci B., Pelosini M., Zuffa E., Facchini L., Imola M., Iori A. P., Proia A., Sica S., Armiento D., Carella A. M., Dellacasa C. M., Fagioli F., Rabusin M., Ferrario A., Elice F., Russo, D, Polverelli, N, Malagola, M, Farina, M, Leoni, A, Bernardi, S, Mammoliti, S, Sacchi, N, Martino, M, Ciceri, F, Zallio, F, Olivieri, A, Falcioni, S, Storti, G, Michieli, M, Carluccio, P, Grassi, A, Oldani, E, Bonifazi, F, Prete, A, Cavattoni, I, Maffeis, M, Pastore, D, Vacca, A, Caravelli, D, Mirabile, M, Mordini, N, Nozzoli, C, Faraci, M, Federico, V, Ronconi, S, Skert, C, Onida, F, Marcatti, M, Piemontese, S, Narni, F, Balduzzi, A, De Simone, G, Picardi, A, De Gobbi, M, Calore, E, Tringali, S, Zecca, M, Secondino, S, Guiducci, B, Pelosini, M, Zuffa, E, Facchini, L, Imola, M, Iori, A, Proia, A, Sica, S, Armiento, D, Carella, A, Dellacasa, C, Fagioli, F, Rabusin, M, Ferrario, A, Elice, F, Russo, D., Polverelli, N., Malagola, M., Farina, M., Leoni, A., Bernardi, S., Mammoliti, S., Sacchi, N., Martino, M., Ciceri, F., Zallio, F., Olivieri, A., Falcioni, S., Storti, G., Michieli, M., Carluccio, P., Grassi, A., Oldani, E., Bonifazi, F., Prete, A., Cavattoni, I. M., Maffeis, M., Pastore, D., Vacca, A., Caravelli, D., Mirabile, M., Mordini, N., Nozzoli, C., Faraci, M., Federico, V., Ronconi, S., Skert, C., Onida, F., Marcatti, M., Piemontese, S., Narni, F., Balduzzi, A., De Simone, G., Picardi, A., De Gobbi, M., Calore, E., Tringali, S., Zecca, M., Secondino, S., Guiducci, B., Pelosini, M., Zuffa, E., Facchini, L., Imola, M., Iori, A. P., Proia, A., Sica, S., Armiento, D., Carella, A. M., Dellacasa, C. M., Fagioli, F., Rabusin, M., Ferrario, A., and Elice, F.

Subjects
Homologous, Myeloid, medicine.medical_specialty, 2019-20 coronavirus outbreak, Bone marrow transplant, Coronavirus disease 2019 (COVID-19), Epidemiology, medicine.medical_treatment, Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), Hematopoietic stem cell transplantation, Acute, Humans, Italy, Pandemics, RNA, Viral, SARS-CoV-2, Stem Cell Transplantation, Transplantation, Homologous, COVID-19, Hematopoietic Stem Cell Transplantation, Leukemia, Myeloid, Acute, Article, hematopoietic stem cell transplantation, covid-19, pandemic, Internal medicine, Pandemic, medicine, Viral, Acute leukemia, Transplantation, Leukemia, business.industry, Hematology, Stem-cell research, RNA, Stem cell, business
Abstract

The Transplant Centers belonging to Gruppo Italiano per il Trapianto di Midollo Osseo (GITMO) conducted a survey with the aim of evaluating the effect of SARS-CoV2 pandemic on the allogeneic transplant activity in Italy. The pandemic period from 1/3/2020 to 31/7/2020 was compared with the same period in 2019. Overall, in 2020 there was a 2.4% reduction in the number of allo-HCT cases compared to 2019. Interestingly, this deflection did not affect the acute leukemia cases (+5.7% in 2020). The use of peripheral blood-derived stem cells (+10.7%) and cryopreservation (97.4% of the centers) was highly adopted in 2020. Despite the sanitary emergency, almost all of the surveyed centers declared no impact of SARS-CoV2 pandemic on the transplant timing and outcomes, and the sanitary policy was positively evaluated by the majority of centers. The emergency measures ensured that only a minority of the allo-HCT patients had been infected by SARS-CoV2; however, a mortality of 42.1% among the allo-HCT patients hospitalized for COVID-19 was recorded. This survey gives us the information that the GITMO Group reacted positively to the pandemic. Thanks to the emergency strategies, the Italian allo-HCT activity continued safely, showing only a minor deflection and offering the same probability of cure to the transplanted patients.

Published
2021

24. Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation

Author

Achini-Gutzwiller, F. R., Snowden, J. A., Corbacioglu, S., Greco, R., Alexander, T., Snowden, J., Badoglio, M., Labopin, M., Abinun, M., Apte, S., Arnold, R., Domenech, A., Brierley, C., Burman, J., Castilla-Llorente, C., Cooper, N., Daghia, G., Daikeler, T., del Papa, N., de Vries-Bouwstra, J., Farge, D., Finke, J., Hagglund, H., Hawkey, C., Henes, J., Hiepe, F., Jessop, H., Kiely, D., Kazmi, M., Kirgizov, K., Kramer, E., Mancardi, G., Marjanovic, Z., Martin, R., Martin, T., Ma, D., Moore, J., Miller, P., Muraro, P., Oliveira, M. -C., Polushin, A., Onida, F., Simoes, B., Puyade, M., Resnick, I., Ricart, E., Rovira, M., Saccardi, R., Saif, M., Sakellari, I., Sharrack, B., Snarski, E., Scherer, H. U., Sossa, C., Withers, B., Wulffraat, N., Zaccara, E., Amrolia, P., Ansari, M., Balduzzi, A., Dalassier, A., Dalle, J. -H., Diaz, C. H., Feuchtinger, T., Locatelli, Franco, Lucchini, G., Galimard, J. -E., Vincent, M. G., Handgretinger, R., Kleinschmidt, K., Lawitschka, A., Martinez, A. P., Peters, C., Rocha, V., Ruggeri, A., Sedlacek, P., Svec, P., Toporski, J., Yesilipek, A., Locatelli F. (ORCID:0000-0002-7976-3654), Achini-Gutzwiller, F. R., Snowden, J. A., Corbacioglu, S., Greco, R., Alexander, T., Snowden, J., Badoglio, M., Labopin, M., Abinun, M., Apte, S., Arnold, R., Domenech, A., Brierley, C., Burman, J., Castilla-Llorente, C., Cooper, N., Daghia, G., Daikeler, T., del Papa, N., de Vries-Bouwstra, J., Farge, D., Finke, J., Hagglund, H., Hawkey, C., Henes, J., Hiepe, F., Jessop, H., Kiely, D., Kazmi, M., Kirgizov, K., Kramer, E., Mancardi, G., Marjanovic, Z., Martin, R., Martin, T., Ma, D., Moore, J., Miller, P., Muraro, P., Oliveira, M. -C., Polushin, A., Onida, F., Simoes, B., Puyade, M., Resnick, I., Ricart, E., Rovira, M., Saccardi, R., Saif, M., Sakellari, I., Sharrack, B., Snarski, E., Scherer, H. U., Sossa, C., Withers, B., Wulffraat, N., Zaccara, E., Amrolia, P., Ansari, M., Balduzzi, A., Dalassier, A., Dalle, J. -H., Diaz, C. H., Feuchtinger, T., Locatelli, Franco, Lucchini, G., Galimard, J. -E., Vincent, M. G., Handgretinger, R., Kleinschmidt, K., Lawitschka, A., Martinez, A. P., Peters, C., Rocha, V., Ruggeri, A., Sedlacek, P., Svec, P., Toporski, J., Yesilipek, A., and Locatelli F. (ORCID:0000-0002-7976-3654)

Abstract

Although modern clinical management strategies have improved the outcome of paediatric patients with severe autoimmune and inflammatory diseases over recent decades, a proportion will experience ongoing or recurrent/relapsing disease activity despite multiple therapies often leading to irreversible organ damage, and compromised quality of life, growth/development and long-term survival. Autologous and allogeneic haematopoietic stem cell transplantation (HSCT) have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children. However, transplant-related outcomes are disease-dependent and long-term outcome data are limited in respect to efficacy and safety. Moreover, balancing risks of HSCT against AD prognosis with continually evolving non-transplant options is challenging. This review appraises published literature on HSCT strategies and outcomes in individual paediatric ADs. We also provide a summary of the European Society for Blood and Marrow Transplantation (EBMT) Registry, where 343 HSCT procedures (176 autologous and 167 allogeneic) have been reported in 326 children (<18 years) for a range of AD indications. HSCT is a promising treatment modality, with potential long-term disease control or cure, but therapy-related morbidity and mortality need to be reduced. Further research is warranted to establish the position of HSCT in paediatric ADs via registries and prospective clinical studies to support evidence-based interspeciality guidelines and recommendations.

Published
2022

25. Myeloablative conditioning with thiotepa-busulfan-fludarabine does not improve the outcome of patients transplanted with active leukemia: final results of the GITMO prospective trial GANDALF-01

Author

Bonifazi, F., Pavoni, C., Peccatori, J., Giglio, F., Arpinati, M., Busca, A., Bernasconi, P., Grassi, A., Iori, A. P., Patriarca, F., Brunello, L., Di Grazia, C., Carella, A. M., Cilloni, D., Picardi, A., Proia, A., Santarone, S., Sorasio, R., Carluccio, P., Chiusolo, Patrizia, Cupri, A., Luppi, M., Nozzoli, C., Baronciani, D., Casini, M., Grillo, G., Musso, M., Onida, F., Palazzo, G., Parma, M., Tringali, S., Vacca, A., Vallisa, D., Sacchi, N., Oldani, E., Masciulli, A., Gheorghiu, A., Girmenia, C., Martino, M., Bruno, B., Rambaldi, A., Ciceri, F., Chiusolo P. (ORCID:0000-0002-1355-1587), Bonifazi, F., Pavoni, C., Peccatori, J., Giglio, F., Arpinati, M., Busca, A., Bernasconi, P., Grassi, A., Iori, A. P., Patriarca, F., Brunello, L., Di Grazia, C., Carella, A. M., Cilloni, D., Picardi, A., Proia, A., Santarone, S., Sorasio, R., Carluccio, P., Chiusolo, Patrizia, Cupri, A., Luppi, M., Nozzoli, C., Baronciani, D., Casini, M., Grillo, G., Musso, M., Onida, F., Palazzo, G., Parma, M., Tringali, S., Vacca, A., Vallisa, D., Sacchi, N., Oldani, E., Masciulli, A., Gheorghiu, A., Girmenia, C., Martino, M., Bruno, B., Rambaldi, A., Ciceri, F., and Chiusolo P. (ORCID:0000-0002-1355-1587)

Abstract

The outcome of refractory/relapsed (R/R) acute leukemias is still dismal and their treatment represents an unmet clinical need. However, allogeneic transplantation (allo-HSCT) remains the only potentially curative approach in this setting. A prospective study (GANDALF-01, NCT01814488; EUDRACT:2012-004008-37) on transplantation with alternative donors had been run by GITMO using a homogeneous myeloablative conditioning regimen with busulfan, thiotepa and fludarabine while GVHD prophylaxis was stratified by donor type. The study enrolled 101 patients; 90 found an alternative donor and 87 ultimately underwent allo-HSCT. Two-year overall survival of the entire and of the transplant population (primary endpoint) were 19% and 22%, without significant differences according to disease, donor type and disease history (relapsed vs refractory patients). Two-year progression-free survival was 19% and 17% respectively. The cumulative incidences of relapse and non-relapse mortality were 49% and 33% at two years. Acute grade II-IV and chronic GVHD occurred in 23 and 10 patients. Dose intensification with a myeloablative two-alkylating regimen as sole strategy for transplanting R/R acute leukemia does seem neither to improve the outcome nor to control disease relapse. A pre-planned relapse prevention should be included in the transplant strategy in this patient population.

Published
2022

26. GITMO Registry Study on Allogeneic Transplantation in Patients Aged ≥60 Years from 2000 to 2017: Improvements and Criticisms

Author

Malagola, M., Polverelli, N., Rubini, V., Martino, M., Patriarca, F., Bruno, B., Giaccone, L., Grillo, G., Bramanti, S., Bernasconi, P., De Gobbi, M., Natale, A., Terruzzi, E., Olivieri, A., Chiusolo, Patrizia, Carella, A. M., Casini, M., Nozzoli, C., Mazza, P., Bassi, S., Onida, F., Vacca, A., Falcioni, S., Luppi, M., Iori, A. P., Pavone, V., Skert, C., Carluccio, P., Borghero, C., Proia, A., Selleri, C., Sacchi, N., Mammoliti, S., Oldani, E., Ciceri, F., Russo, D., Bonifazi, F., Chiusolo P. (ORCID:0000-0002-1355-1587), Malagola, M., Polverelli, N., Rubini, V., Martino, M., Patriarca, F., Bruno, B., Giaccone, L., Grillo, G., Bramanti, S., Bernasconi, P., De Gobbi, M., Natale, A., Terruzzi, E., Olivieri, A., Chiusolo, Patrizia, Carella, A. M., Casini, M., Nozzoli, C., Mazza, P., Bassi, S., Onida, F., Vacca, A., Falcioni, S., Luppi, M., Iori, A. P., Pavone, V., Skert, C., Carluccio, P., Borghero, C., Proia, A., Selleri, C., Sacchi, N., Mammoliti, S., Oldani, E., Ciceri, F., Russo, D., Bonifazi, F., and Chiusolo P. (ORCID:0000-0002-1355-1587)

Abstract

Today, allogeneic stem cell transplantation (allo-SCT) can be offered to patients up to age 70 to 72 years and represents one of the most effective curative treatments for many hematologic malignancies. The primary objective of the study was to collect data from the allo-SCTs performed in Italy between 2000 and 2017 in patients aged ≥60 years to evaluate the changes in safety and efficacy outcomes, as well as their distribution and characteristics over time. The Italian Group for Bone Marrow Transplantation, Hematopoietic Stem Cells and Cell Therapy (GITMO) AlloEld study (ClinicalTrials.gov identifier NCT04469985) is a retrospective analysis of allo-SCTs performed at 30 Italian transplantation centers in older patients (age ≥60 years) between 2000 and 2017 (n = 1996). For the purpose of this analysis, patients were grouped into 3 time periods: time A, 2000 to 2005 (n = 256; 12%); time B, 2006 to 2011 (n = 584; 29%); and time C, 2012 to 2017 (n = 1156; 59%). After a median follow-up of 5.6 years, the 5-year nonrelapse mortality (NRM) remained stable (time A, 32.8%; time B, 36.2%; and time C, 35.0%; P = .5), overall survival improved (time A, 28.4%; time B, 31.8%; and time C, 37.3%; P = .012), and the cumulative incidence of relapse was reduced (time A, 45.3%; time B, 38.2%; time C, 30.0%; P < .0001). The 2-year incidence of extensive chronic graft-versus-host disease was reduced significantly (time A, 17.2%; time B, 15.8%; time C, 12.2%; P = .004). Considering times A and B together (2000 to 2011), the 2-year NRM was positively correlated with the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) score; NRM was 25.2% in patients with an HCT-CI score of 0, 33.9% in those with a score of 1 or 2, and 36.1% in those with a score of 3 (P < .001). However, after 2012, the HCT-CI score was not significantly predictive of NRM. This study shows that the transplantation procedure in elderly patients became more effective over time. Relapse incidence remains t

Published
2022

27. Haplotype motif-based models for KIR-genotype informed selection of hematopoietic cell donors fail to predict outcome of patients with Myelodysplastic Syndromes or secondary Acute Myeloid Leukemia (vol 11,& nbsp;584520, 2021)

Author

Schetelig, J., Baldauf, H., Koster, L., Kuxhausen, M., Heidenreich, F., Wreede, L.C. de, Spellman, S., Gelder, M. van, Bruno, B., Onida, F., Lange, V., Massalski, C., Potter, V., Ljungman, P., Schaap, N., Hayden, P., Lee, S.P.N.J., Kroeger, N., Hsu, K.T.Y., Schmidt, A.H., Yakoub-Agha, I., and Robin, M.

Subjects
KIR3DL1, unrelated donor, hematopoietic stem cell transplantation, donor selection, KIR2DS1, KIR
Published
2021

28. Autologous stem cell transplantation for progressive systemic sclerosis: a prospective non-interventional study from the European Society for Blood and Marrow Transplantation Autoimmune Disease Working Party

Author

Henes, J., Oliveira, M.C., Labopin, M., Badoglio, M., Scherer, H.U., Papa, N. del, Daikeler, T., Schmalzing, M., Schroers, R., Martin, T., Pugnet, G., Simoes, B., Michonneau, D., Marijt, E.W.A., Lioure, B., Bay, J.O., Snowden, J.A., Rovira, M., Huynh, A., Onida, F., Kanz, L., Marjanovic, Z., Farge, D., NISSC1 Members, University of São Paulo (USP), CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Centre International des greffes [CHU Saint-Antoine] (EBMT), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Saint-Antoine [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU), Leiden University Medical Center (LUMC), University of Basel (Unibas), CHU Strasbourg, Hôpital Purpan [Toulouse], CHU Toulouse [Toulouse], Hopital Saint-Louis [AP-HP] (AP-HP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), CHU Clermont-Ferrand, Institut Universitaire du Cancer de Toulouse - Oncopole (IUCT Oncopole - UMR 1037), Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-CHU Toulouse [Toulouse]-Institut National de la Santé et de la Recherche Médicale (INSERM), and Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Subjects
Adult, medicine.medical_specialty, Transplantation Conditioning, Cyclophosphamide, [SDV]Life Sciences [q-bio], medicine.medical_treatment, Aucun, Hematopoietic stem cell transplantation, Transplantation, Autologous, MESH: Scleroderma, Systemic, Article, Autoimmune Diseases, 03 medical and health sciences, 0302 clinical medicine, Autologous stem-cell transplantation, Bone Marrow, MESH: Autoimmune Diseases, Internal medicine, Clinical endpoint, Humans, Medicine, Prospective Studies, Progression-free survival, MESH: Hematopoietic Stem Cell Transplantation, MESH: Transplantation Conditioning, Aged, MESH: Aged, 030203 arthritis & rheumatology, MESH: Humans, Scleroderma, Systemic, business.industry, Hazard ratio, Hematopoietic Stem Cell Transplantation, MESH: Cyclophosphamide, MESH: Adult, Hematology, TRANSPLANTE AUTÓLOGO, MESH: Transplantation, Autologous, MESH: Prospective Studies, 3. Good health, Transplantation, MESH: Scleroderma, Diffuse, 030220 oncology & carcinogenesis, Scleroderma, Diffuse, MESH: Bone Marrow, Stem cell, business, medicine.drug
Abstract

Three randomized controlled trials in early severe systemic sclerosis demonstrated that autologous hematopoietic stem cell transplantation was superior to standard cyclophosphamide therapy. This European Society for Blood and Marrow Transplantation multicenter, prospective, non-interventional study was designed to further decipher efficacy and safety of this procedure for severe systemic sclerosis patients in real-life practice and to search for prognostic factors. All consecutive adult patients with systemic sclerosis undergoing a first autologous hematopoietic stem cell transplant between December 2012 and February 2016 were prospectively included in the study. The primary endpoint was progression-free survival. Secondary endpoints were overall survival, non-relapse mortality, response and incidence of progression. Eighty patients with systemic sclerosis were included. The median duration of the follow-up was 24 (range, 6-57) months after stem cell transplantation using cyclophosphamide plus antithymocyte globulin conditioning for all, with CD34+ selection in 35 patients. At 2 years, the progression- free survival rate was 81.8%, the overall survival rate was 90%, the response rate was 88.7% and the incidence of progression was 11.9%. The 100-day non-relapse mortality rate was 6.25% (n=5) with four deaths from cardiac events, including three due to cyclophosphamide toxicity. Modified Rodnan skin score and forced vital capacity improved with time (P24 and older age at transplantation were associated with lower progression-free survival (hazard ratios 3.32 and 1.77, respectively). CD34+-cell selection was associated with better response (hazard ratio 0.46). This study confirms the efficacy of autologous stem cell transplantation, using nonmyeloablative conditioning, in real-life practice for severe systemic sclerosis. Careful cardio-pulmonary assessment to identify organ involvement at the time of the patient’s referral, reduced cyclophosphamide doses and CD34+-cell selection may improve outcomes. The study was registered at ClinicalTrials.gov: NCT02516124.

Published
2021
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29. Recent Advancements in Hematology: Knowledge, Methods and Dissemination, Part 2

Author

Corre, J, Sabbah, M, Schjesvold, F, Zeidan, AM, Buccisano, F, Sallman, D, Mazzucato, M, Madden, LA, Martini, M, Van Breda, E, Dolcetti, R, Busca, A, Cook, G, Onida, F, Versari, A, Kiladjian, J-J, Walter, RB, Garderet, L, Robin, M, Signore, A, Corre, J, Sabbah, M, Schjesvold, F, Zeidan, AM, Buccisano, F, Sallman, D, Mazzucato, M, Madden, LA, Martini, M, Van Breda, E, Dolcetti, R, Busca, A, Cook, G, Onida, F, Versari, A, Kiladjian, J-J, Walter, RB, Garderet, L, Robin, M, and Signore, A

Abstract

Recent Advancements in Hematology: Knowledge, Methods and Dissemination is a series of commentary article which is published on a biannual basis by the editorial board of the journal Hemato [...]

Published
2021

36. Autologous haematopoietic stem cell transplantation without CD34+ cell selection in refractory Crohn's disease

Author

Cassinotti, A., Annaloro, C., Ardizzone, S., Onida, F., Della Volpe, A., Clerici, M., Usardi, P., Greco, S., Maconi, G., Porro, G.. Bianchi, and Deliliers, G. Lambertenghi

Subjects
Autografts -- Methods, Autografts -- Research, CD4 lymphocytes -- Identification and classification, CD4 lymphocytes -- Research, Crohn's disease -- Care and treatment, Hematopoietic stem cells -- Transplantation, Hematopoietic stem cells -- Usage, Hematopoietic stem cells -- Research, Health
Published
2008

37. Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases

Author

Sharrack, B., Saccardi, R., Alexander, T., Badoglio, M., Burman, J., Farge, D., Greco, R., Jessop, H., Kazmi, M., Kirgizov, K., Labopin, M., Mancardi, G., Martin, R., Moore, J., Muraro, P.A., Rovira Tarrats, Montserrat, Sormani, M.P., Snowden, J.A., Snowden, J., McGrath, E., Bambi, F., Sanchez-Guijo, F., Worel, N., Badolglio, M., Abinun, M., Arnold, R., Brierley, C., Castilla-Llorente, C., Cooper, N., Daikeler, T., del Papa, N., Finke, J., Hagglund, H., Henes, J., Hiepe, F., Kiely, D., Marjanovic, Z., Martin, T., Ma, D., Miller, P., Muraro, P., Oliveira, M.C., Polushin, A., Onida, F., Simoes, B., Puyade, M., Resnick, I., Rovira, Montserrat, Saif, M., Sakellari, I., Snarski, E., Scherer, H.U., Sossa, C., de Vries-Bouwstra, J., Wulffraat, N., Zaccara, E., Universitat Autònoma de Barcelona, University of Zurich, and Snowden, John A

Subjects
Feature, medicine.medical_specialty, Multiple Sclerosis, Neurologi, Bone marrow transplantation, 2747 Transplantation, medicine.medical_treatment, Immunology, 2720 Hematology, Chronic inflammatory demyelinating polyneuropathy, 610 Medicine & health, Disease, Hematopoietic stem cell transplantation, Transplantation, Autologous, Accreditation, Autoimmune Diseases, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, medicine, Humans, European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT) and EBMT (JACIE), Intensive care medicine, Peripheral neuropathies, Transplantation, Hematology, Neuromyelitis optica, business.industry, Multiple sclerosis, Hematopoietic Stem Cell Transplantation, 1103 Clinical Sciences, medicine.disease, 10040 Clinic for Neurology, Clinical trial, Europe, surgical procedures, operative, Neurology, business, 030217 neurology & neurosurgery, 030215 immunology
Abstract

These updated EBMT guidelines review the clinical evidence, registry activity and mechanisms of action of haematopoietic stem cell transplantation (HSCT) in multiple sclerosis (MS) and other immune-mediated neurological diseases and provide recommendations for patient selection, transplant technique, follow-up and future development. The major focus is on autologous HSCT (aHSCT), used in MS for over two decades and currently the fastest growing indication for this treatment in Europe, with increasing evidence to support its use in highly active relapsing remitting MS failing to respond to disease modifying therapies. aHSCT may have a potential role in the treatment of the progressive forms of MS with a significant inflammatory component and other immune-mediated neurological diseases, including chronic inflammatory demyelinating polyneuropathy, neuromyelitis optica, myasthenia gravis and stiff person syndrome. Allogeneic HSCT should only be considered where potential risks are justified. Compared with other immunomodulatory treatments, HSCT is associated with greater short-term risks and requires close interspeciality collaboration between transplant physicians and neurologists with a special interest in these neurological conditions before, during and after treatment in accredited HSCT centres. Other experimental cell therapies are developmental for these diseases and patients should only be treated on clinical trials.

Published
2020

38. Special considerations in the management of adult patients with acute leukaemias and myeloid neoplasms in the COVID-19 era: recommendations from a panel of international experts

Author

Zeidan, AM, Boddu, PC, Patnaik, MM, Bewersdorf, JP, Stahl, M, Rampal, RK, Shallis, RM, Steensma, DP, Savona, MR, Sekeres, MA, Roboz, GJ, DeAngelo, DJ, Schuh, AC, Padron, E, Zeidner, JF, Walter, RB, Onida, F, Fathi, AT, DeZern, A, Hobbs, G, Stein, EM, Vyas, P, Wei, AH, Bowen, DT, Montesinos, P, Griffiths, EA, Verma, AK, Keyzner, A, Bar-Natan, M, Navada, SC, Kremyanskaya, M, Goldberg, AD, Al-Kali, A, Heaney, ML, Nazha, A, Salman, H, Luger, S, Pratz, KW, Konig, H, Komrokji, R, Deininger, M, Cirici, BX, Bhatt, VR, Silverman, LR, Erba, HP, Fenaux, P, Platzbecker, U, Santini, V, Wang, ES, Tallman, MS, Stone, RM, and Mascarenhas, J

Abstract

The ongoing COVID-19 pandemic caused by severe acute respiratory syndrome coronavirus 2 is a global public health crisis. Multiple observations indicate poorer post-infection outcomes for patients with cancer than for the general population. Herein, we highlight the challenges in caring for patients with acute leukaemias and myeloid neoplasms amid the COVID-19 pandemic. We summarise key changes related to service allocation, clinical and supportive care, clinical trial participation, and ethical considerations regarding the use of lifesaving measures for these patients. We recognise that these recommendations might be more applicable to high-income countries and might not be generalisable because of regional differences in health-care infrastructure, individual circumstances, and a complex and highly fluid health-care environment. Despite these limitations, we aim to provide a general framework for the care of patients with acute leukaemias and myeloid neoplasms during the COVID-19 pandemic on the basis of recommendations from international experts.

Published
2020

39. Recent Advancements in Hematology: Knowledge, Methods and Dissemination, Part 1

Author

Fitzgibbon, J, Park, S, Cook, G, Paiva, B, Gloghini, A, Van Breda, E, Busti, F, Garderet, L, Dolcetti, R, Robin, M, Martino, R, Busca, A, Sabbah, M, De Rosa, S, Martini, M, Onida, F, Aucouturier, P, Schjesvold, F, Minvielle, S, Mazzucato, M, Terragna, C, Delforge, M, Harrison, C, Carbone, A, Fitzgibbon, J, Park, S, Cook, G, Paiva, B, Gloghini, A, Van Breda, E, Busti, F, Garderet, L, Dolcetti, R, Robin, M, Martino, R, Busca, A, Sabbah, M, De Rosa, S, Martini, M, Onida, F, Aucouturier, P, Schjesvold, F, Minvielle, S, Mazzucato, M, Terragna, C, Delforge, M, Harrison, C, and Carbone, A

Abstract

Recent Advancements in Hematology: Knowledge, Methods and Dissemination is a series of editorials which is published on a biannual basis by the editorial board of the journal Bloods [...]

Published
2020

40. Integrated Genomic, Functional, and Prognostic Characterization of Atypical Chronic Myeloid Leukemia

Author

Fontana, D, Ramazzotti, D, Aroldi, A, Redaelli, S, Magistroni, V, Pirola, A, Niro, A, Massimino, L, Mastini, C, Brambilla, V, Bombelli, S, Bungaro, S, Morotti, A, Rea, D, Stagno, F, Martino, B, Campiotti, L, Caocci, G, Usala, E, Merli, M, Onida, F, Bregni, M, Elli, E, Fumagalli, M, Ciceri, F, Perego, R, Pagni, F, Mologni, L, Piazza, R, Gambacorti-Passerini, C, Fontana, Diletta, Ramazzotti, Daniele, Aroldi, Andrea, Redaelli, Sara, Magistroni, Vera, Pirola, Alessandra, Niro, Antonio, Massimino, Luca, Mastini, Cristina, Brambilla, Virginia, Bombelli, Silvia, Bungaro, Silvia, Morotti, Alessandro, Rea, Delphine, Stagno, Fabio, Martino, Bruno, Campiotti, Leonardo, Caocci, Giovanni, Usala, Emilio, Merli, Michele, Onida, Francesco, Bregni, Marco, Elli, Elena Maria, Fumagalli, Monica, Ciceri, Fabio, Perego, Roberto A, Pagni, Fabio, Mologni, Luca, Piazza, Rocco, Gambacorti-Passerini, Carlo, Fontana, D, Ramazzotti, D, Aroldi, A, Redaelli, S, Magistroni, V, Pirola, A, Niro, A, Massimino, L, Mastini, C, Brambilla, V, Bombelli, S, Bungaro, S, Morotti, A, Rea, D, Stagno, F, Martino, B, Campiotti, L, Caocci, G, Usala, E, Merli, M, Onida, F, Bregni, M, Elli, E, Fumagalli, M, Ciceri, F, Perego, R, Pagni, F, Mologni, L, Piazza, R, Gambacorti-Passerini, C, Fontana, Diletta, Ramazzotti, Daniele, Aroldi, Andrea, Redaelli, Sara, Magistroni, Vera, Pirola, Alessandra, Niro, Antonio, Massimino, Luca, Mastini, Cristina, Brambilla, Virginia, Bombelli, Silvia, Bungaro, Silvia, Morotti, Alessandro, Rea, Delphine, Stagno, Fabio, Martino, Bruno, Campiotti, Leonardo, Caocci, Giovanni, Usala, Emilio, Merli, Michele, Onida, Francesco, Bregni, Marco, Elli, Elena Maria, Fumagalli, Monica, Ciceri, Fabio, Perego, Roberto A, Pagni, Fabio, Mologni, Luca, Piazza, Rocco, and Gambacorti-Passerini, Carlo

Abstract

Atypical chronic myeloid leukemia (aCML) is a BCR-ABL1-negative clonal disorder, which belongs to the myelodysplastic/myeloproliferative group. This disease is characterized by recurrent somatic mutations in SETBP1, ASXL1 and ETNK1 genes, as well as high genetic heterogeneity, thus posing a great therapeutic challenge. To provide a comprehensive genomic characterization of aCML we applied a high-throughput sequencing strategy to 43 aCML samples, including both whole-exome and RNA-sequencing data. Our dataset identifies ASXL1, SETBP1, and ETNK1 as the most frequently mutated genes with a total of 43.2%, 29.7 and 16.2%, respectively. We characterized the clonal architecture of 7 aCML patients by means of colony assays and targeted resequencing. The results indicate that ETNK1 variants occur early in the clonal evolution history of aCML, while SETBP1 mutations often represent a late event. The presence of actionable mutations conferred both ex vivo and in vivo sensitivity to specific inhibitors with evidence of strong in vitro synergism in case of multiple targeting. In one patient, a clinical response was obtained. Stratification based on RNA-sequencing identified two different populations in terms of overall survival, and differential gene expression analysis identified 38 significantly overexpressed genes in the worse outcome group. Three genes correctly classified patients for overall survival.

Published
2020

47. Bortezomib (Velcade)-thalidomide-dexamethasone is superior to thalidomide-dexamethasone in patients with multiple myeloma progressing or relapsing after autologous transplantation: 0117

Author

Garderet, L., Iacobelli, S., Moreau, P., Dib, M., Caillot, D., Niederwieser, D., Masszi, T., Fontan, J., Michallet, M., Gratwohl, A., Milone, G., Doyen, C., Pegourie, B., Hajek, R., Casassus, P., Kolb, B., Chaleteix, C., Hertenstein, B., Onida, F., Ludwig, H., Vekemans, M. C., Ketterer, N., Daguenel, A., Koenecke, C., Gorin, N.-C., Harousseau, J.-L., de Witte, T., Morris, C., and Gahrton, G.

Published
2011

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