13 results on '"Iacobelli, Simona"'
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2. Additional file 1 of Analyzing differences between restricted mean survival time curves using pseudo-values
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Ambrogi, Federico, Iacobelli, Simona, and Andersen, Per Kragh
- Abstract
Additional file 1 An illustrative example with different methods for RMST estimation.
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- 2022
- Full Text
- View/download PDF
3. Role of Age and Hematopoietic Cell Transplantation-Specific Comorbidity Index in Myelodysplastic Patients Undergoing an Allotransplant: A Retrospective Study from the Chronic Malignancies Working Party of the European Group for Blood and Marrow Transplantation
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Carré, Martin, Porcher, Raphaël, Finke, Jürgen, Ehninger, Gerhard, Koster, Linda, Beelen, Dietrich, Ganser, Arnold, Volin, Liisa, Lozano, Sara, Friis, Lone, Michallet, Mauricette, Tischer, Johanna, Olavarria, Eduardo, Cascon, Maria Jesús Pascual, Iacobelli, Simona, Koc, Yener, Jindra, Pavel, Arat, Mutlu, de Witte, Theo, Yakoub Agha, Ibrahim, Kröger, Nicolaus, Robin, Marie, and Agha, Ibrahim Yakoub
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Adult ,medicine.medical_specialty ,Transplantation Conditioning ,Multivariate analysis ,Cancer development and immune defence Radboud Institute for Molecular Life Sciences [Radboudumc 2] ,medicine.medical_treatment ,Myelodysplastic syndromes ,Medizin ,Comorbidity ,Hematopoietic stem cell transplantation ,Comorbidities ,03 medical and health sciences ,All institutes and research themes of the Radboud University Medical Center ,Age ,0302 clinical medicine ,Bone Marrow ,Neoplasms ,hemic and lymphatic diseases ,Internal medicine ,medicine ,Humans ,Retrospective Studies ,Transplantation ,Nonrelapse mortality ,business.industry ,Hazard ratio ,Hematopoietic Stem Cell Transplantation ,Retrospective cohort study ,Hematology ,Settore MED/15 ,medicine.disease ,Settore MED/01 ,030220 oncology & carcinogenesis ,Stem cell ,business ,030215 immunology - Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only potentially curative option for myelodysplastic syndromes (MDSs) but is severely limited by nonrelapse mortality (NRM), especially in this mostly older population. Comorbidity assessment is crucial to predict NRM and often assessed with the Hematopoietic Cell Transplantation-Specific Comorbidity Index (HCT-CI). Moreover, the impact of age on NRM still remains a matter of debate. In recent years, the age at which transplants are made has been progressively increasing, and patients with comorbidities have become more common. Extricating the respective roles of age and comorbidities in toxic mortality is all the more important. This study by the European Group for Blood and Marrow Transplantation registry included 1245 adult patients who underwent a first allogeneic stem cell transplantation for MDSs between 2003 and 2014. Overall, 4-year NRM and overall survival were 32% and 47%, respectively. When considered as continuous predictors, HCT-CI score and age were associated with an increased hazard ratio (HR) for NRM. In multivariate analysis, age band (HR, 1.13; 95% CI, 1.02 to 1.25; P= .016), HCT-CI ≥3 (HR, 1.34; 95% CI, 1.04 to 1.73; P = .022), and Karnofsky Performance Status ≤80 (HR, 2.03; 95% CI, 1.52 to 2.73; P.0001) were significantly predictive of a worse NRM. In our large cohort, both comorbidities, evaluated by the original HCT-CI score, and chronological age significantly affected NRM. Thus, age should be part of the transplant decision-making process and should be integrated in future scoring systems predicting outcomes of HSCT in MDSs.
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- 2020
4. Centre characteristics and procedure-related factors have an impact on outcomes of allogeneic transplantation for patients with CLL: a retrospective analysis from the European Society for Blood and Marrow Transplantation (EBMT)
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Schetelig, Johannes, de Wreede, Liesbeth C., Andersen, Niels S., Moreno, Carol, van Gelder, Michel, Vitek, Antonin, Karas, Michal, Michallet, Mauricette, Machaczka, Maciej, Gramatzki, Martin, Beelen, Dietrich, Finke, Jürgen, Delgado, Julio, Volin, Liisa, Passweg, Jakob, Dreger, Peter, Schaap, Nicolaas, Wagner, Eva, Henseler, Anja, van Biezen, Anja, Bornhäuser, Martin, Iacobelli, Simona, Putter, Hein, Schönland, Stefan O., Kröger, Nicolaus, Esteve, Jordi, Ljungman, Per, de Witte, Theo, Stelljes, Matthias, Sierra, Jorge, Socié, Gerard, Ganser, Arnold, Wulf, Gerhard G., Deconinck, Eric, Faber, Edgar, Feguex, Nathalie, Gedde-Dahl, Tobias, Kolbe, Karin, Chalandon, Yves, Krüger, William, Huynh, Anne, Bourhis, Jean Henri, Schouten, Harry, Ribera Santasusana, Josep M., Interne Geneeskunde, MUMC+: MA Hematologie (9), RS: GROW - R3 - Innovative Cancer Diagnostics & Therapy, and Chalandon, Yves
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Oncology ,Male ,Transplantation Conditioning ,Cancer development and immune defence Radboud Institute for Molecular Life Sciences [Radboudumc 2] ,Chronic lymphocytic leukemia ,Medizin ,QUALITY MANAGEMENT-SYSTEM ,Kaplan-Meier Estimate ,risk factor analysis ,GUIDELINES ,Biochemistry ,allogeneic stem cell transplantation ,centre effects ,chronic lymphocytic leukaemia ,frailties ,Transplantation Conditioning / methods ,0302 clinical medicine ,Recurrence ,Risk Factors ,Leukemia, Lymphocytic, Chronic, B-Cell / mortality ,Registries ,Delivery of Health Care / statistics & numerical data ,IBRUTINIB ,ddc:616 ,0303 health sciences ,ALEMTUZUMAB ,Hazard ratio ,Hematopoietic Stem Cell Transplantation ,Professional Practice ,Hematology ,Middle Aged ,3. Good health ,Europe ,030220 oncology & carcinogenesis ,ABT-199 ,Cohort ,SURVIVAL ,Alemtuzumab ,Female ,Europe / epidemiology ,medicine.drug ,Adult ,medicine.medical_specialty ,Allogeneic transplantation ,Immunology ,Lower risk ,DIAGNOSIS ,Settore MED/01 - Statistica Medica ,03 medical and health sciences ,Leukemia, Lymphocytic, Chronic, B-Cell / therapy ,Hematopoietic Stem Cell Transplantation / methods ,Internal medicine ,medicine ,Humans ,Karnofsky Performance Status ,030304 developmental biology ,Aged ,Retrospective Studies ,business.industry ,Proportional hazards model ,CHRONIC LYMPHOCYTIC-LEUKEMIA ,Retrospective cohort study ,STEM-CELL TRANSPLANTATION ,Cell Biology ,medicine.disease ,Leukemia, Lymphocytic, Chronic, B-Cell ,Confidence interval ,Surgery ,Professional Practice / statistics & numerical data ,Transplantation ,business ,Delivery of Health Care ,030215 immunology ,RESPONSES - Abstract
Introduction:Even in the era of novel targeted therapies for the treatment of Chronic Lymphocytic Leukemia (CLL) patients, such as BTK, PI3K and BCL2 inhibitors, allogeneic hematopoietic stem cell transplantations (alloHCT) will remain an important treatment option for a subset of patients with very high risk CLL. The current study focused on the impact of center and procedure-related factors on outcomes after alloHCT, taking into account the impact of patient- and disease-related risk factors. Patients and Methods:Data of 684 CLL patients who received a first alloHCT between 2000 and 2011 were analyzed. Their data were collected as part of the EBMT CLL Data Quality Initiative. Outcomes of interest were Event-Free Survival (EFS) up to 5 years after transplantation and mortality in the first 100 days after alloHCT. Outcomes were analyzed by means of the Kaplan-Meier method and Cox proportional hazards models with a frailty (random effects) component to take into account unexplained center heterogeneity. The following factors describing center characteristics or the transplant procedure were analyzed: experience in alloHCT in general and, for CLL specifically, accreditation by the Joint Accreditation Committee-ISCT & EBMT (JACIE), Gross National Income (GNI)/capita based on purchasing power parity (PPP) (GNI/cap), donor type, donor-patient sex-match, type of conditioning, stem cell source and T-cell depletion (TCD). Results:Five-year EFS of the whole cohort was 37% (95% Confidence Interval, 33%-42%), Day-100 survival was 90% (88%-92%). Experience of the transplant center was measured by the number of all alloHCTs, and alloHCTs for patients with CLL respectively. The median total number of alloHCTs per center per year was 45 (range 0-169) and the median number of CLL alloHCTs was only 2 per center per year (range 0-19). Greater experience with transplantation of patients with CLL (Hazard Ratio (HR) 0.96 per additional transplant, p=0.002), JACIE accreditation (HR 0.7, p=0.045) and a higher GNI/cap (HR 0.4, 95% CI 0.2-0.96, p=0.04) showed a protective impact on 5-year EFS in the Cox model. In vivo TCD with alemtuzumab (HR 1.5 compared to no TCD, p=0.03) and a female donor for a male patient (HR 1.4 compared to a male donor for a male patient, p=0.02) were the only procedure-related factors significantly associated with EFS. Event-Free Survival after in vivo TCD with Anti-Thymocyte-Globulin or after ex vivo TCD was comparable to EFS without TCD (HR 0.9, 0.7-1.3, p=0.6; HR 0.9, 0.5-1.6, p=0.8). Non-myeloablative conditioning did not have a negative impact on 5-year EFS, and exposed patients to a lower risk of non-relapse mortality. Measured and unmeasured center characteristics did not have a significant impact on 100-day mortality. Even when correcting for patient-, procedure- and center-related characteristics, there was still significant variation in center outcome, expressed by center-specific HRs derived from the frailty models, ranging from 0.6 to 1.2. Their impact is illustrated in a model-based plot for EFS (see Figure) which shows outcomes for three reference patients with the same characteristics who would be transplanted in three centers with the same measured characteristics but with the highest, average and lowest HRs in the dataset. These unexplained center effects likely represent a mixture of differences which could apply to the location of the transplant center, unmeasured characteristics of the patient population transplanted at this center, selection criteria which were not reported and factors determining the success of the transplant procedure which might differ between centers. Conclusion: We have confirmed that both center- and procedure-related factors have a significant impact on the EFS of patients with CLL undergoing alloHCT. Our results may help to interpret outcomes of single or multicenter studies better. Since non-myeloablative conditioning did not have a negative impact on EFS and exposed patients to a lower risk of non-relapse mortality, this approach should be favored for future alloHCT for CLL. Probability of Event-Free Survival up to Five Years Post-HCT for three Reference Patients Contribution: J.S. designed the research and wrote the paper. L.C.d.W conducted the statistical analysis and produced the figure. Figure Figure. Disclosures Schetelig: Sanofi: Honoraria. Gramatzki:Janssen: Other: Travel/Accommodation/Expenses, Research Funding. Dreger:Gilead: Consultancy; Gilead: Speakers Bureau; Janssen: Consultancy; Novartis: Speakers Bureau; Novartis: Consultancy; Roche: Consultancy.
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- 2017
5. Unusual meteorological phenomena: Vallombrosa forest windthrow caused by the storm of March 5th, 2015. Analyses and evaluations
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CIBECCHINI, DANIELE, AMINTI, GIOVANNI, ANTONELLO, LEONARDO, BRACALINI, MATTEO, CAMBI, MARTINA, CARRARI, ELISA, CROCI, FRANCESCO, ERRICO, ALESSANDRO, FODERI, CRISTIANO, FRASSINELLI, NICCOLO', GIAMBASTIANI, YAMUNA, GIANNETTI, FRANCESCA, IACOBELLI, SIMONA, LASCHI, ANDREA, RACANELLI, VERONICA, SASSOLI, MARTINA, and MIGLIORINI, DUCCIO
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Catastrophic meteorological events, Windstorm, Global change, Forest stability, Forest Management, Windthrows - Published
- 2015
6. Equestrian pilgrim along the Francigena way. Planning, surveying and testing Francigena as an equestrian path in Tuscany
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BAMBI, GIANLUCA, IACOBELLI, SIMONA, MONTI, MASSIMO, and BARBARI, MATTEO
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Francigena Way, equestrian tourism, horse paths, horse building - Published
- 2015
7. Comparison of Haematopoietic Stem Cell Transplantation Approaches in Primary Plasma Cell Leukaemia
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William Arcese, Keith Wilson, Linda Koster, Paolo Bartolomeo, Eefke Petersen, Jan J. Cornelissen, Patrice Chevallier, Nicolaus Kröger, Bruno Lioure, Curly Morris, Stig Lenhoff, Sarah Lawless, Xavier Poiré, Nicolaas Schaap, Reza Tabrizi, Roberto Foa, Laurent Garderet, Iacobelli Simona, Martin R. Schipperus, John A. Snowden, Didier Blaise, Stefan Schönland, Ghulam J. Mufti, Anja van Biezen, Nigel H. Russell, and Jürgen Finke
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Cancer Research ,medicine.medical_specialty ,Allogeneic transplantation ,business.industry ,medicine.medical_treatment ,Hazard ratio ,Immunology ,Hematopoietic stem cell transplantation ,Cell Biology ,Hematology ,Biochemistry ,Transplantation ,03 medical and health sciences ,0302 clinical medicine ,Autologous stem-cell transplantation ,Oncology ,Median follow-up ,030220 oncology & carcinogenesis ,Internal medicine ,medicine ,Clinical endpoint ,Progression-free survival ,business ,030215 immunology - Abstract
Introduction Primary plasma cellleukemiais a rare and aggressive disorder. While autologous stem cell transplantation has been shown to improve outcome, response durations are short and further strategies are needed. Data is needed to help guide clinicians on the best approach to manage this disease. Therefore in an era of novel agents and improved autologous and allogeneic transplantation strategies this study compared different transplantation approaches in the treatment of PCL. Material and Methods A retrospective analysis was undertaken of theEuropean Group for Bone Marrow Transplantation (EBMT) experience of patients with primary PCL undergoing hematopoietic stem cell transplantation between 1998 and 2012. Only patients who had achieved complete response, partial response or stable disease prior to transplantation were included. Patients with progressive or relapsed disease were excluded. Data was collected using MED A and MED B forms. The primary end point was overall survival and data was analysed according to the information on the planned transplant strategy reported at the time of first transplant (intent-to-treat principle). Results A total of 460 patients were identified and categorised into 4 transplant groups. 1. One patient did not proceed to Allo 2. Four patients not classified The follow up period ranged from 1 to 208 months with a median follow up of 48.9 months. Patients undergoing allo upfront were found to have the worst overall survival. Compared to single auto this was statistically significant with a Hazard Ratio (HR) 1.85 (p=0.007). Compared to auto-allo the allo up front group had an even higher risk HR 3.18 (p=0.018). The double auto versus single auto had a HR 1.39 which was not statistically significant (p=0.28). The auto-allo compared to single auto has a time varying effect with a higher mortality at the beginning and better risk afterwards. The effect on average is not significant HR 0.58 (p=0.24). When we consider time varying effects three periods were examined, 0-12, 12-36 and 36-60 months. Allo upfront is still confirmed to be inferior to single auto and also to auto-allo in the first year. Auto-allo has a trend to superiority versus single or double auto in the mid-term. However due to lack of follow up data in the auto-allo group it is difficult to assess possible benefits of auto-allo in the long term as there are few patients still at risk. Our initial analysis does suggest an advantage. Preliminary analysis suggests that the auto-allo group enjoy the best progression free survival. The superiority of auto-allo versus allo upfront may be attributed to the higher rates of non-relapse related mortality observed in allo upfront. Conclusion Our preliminary work has demonstrated that allo upfront is associated with the worst overall survival. Further data is needed to reliably assess the outcome of auto-allo group which appears to be superior. Therefore we plan to request additional information from involved centres to strengthen the power of our results. Table Table. Disclosures Foà: Ariad: Speakers Bureau; Pfizer: Speakers Bureau; BMS: Consultancy; Celgene: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Gilead: Consultancy, Speakers Bureau; Janssen-Cilag: Consultancy, Speakers Bureau; Genetech: Consultancy; Roche: Consultancy, Speakers Bureau. Garderet:Takeda: Consultancy; Amgen: Consultancy; BMS: Consultancy, Honoraria; Novartis: Consultancy.
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- 2016
8. The role of renal impairment at diagnosis in multiple myeloma undergoing autologous transplantation. A retrospective analysis of the CMWP
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Scheid, Christof, Hoek, Jennifer, Russell, Nigel H., Didier Blaise, Mayer, Jiri, Ljungman, Per, Lenhoff, Stig, Kobbe, Guido, Iacobelli, Simona, Isaksson, Cecilia, Schaap, Nicolaas, Basak, Grzegorz, Hayden, Patrick, Gonzalez Muniz, Soledad, Apperley, Jane, Rambaldi, Alessandro, Wilson, Keith M. O., Parody Porras, Rocio, Jantunen, Esa, Anagnostopoulos, Achilles, Schoenland, Stefan, Yakoub-Agha, Ibrahim, Kroeger, Nicolaus, Morris, Curly, and Garderet, Laurent
9. Pre-engraftment and post-engraftment Gram=negative rods bacteremia in HSCT patients: Risk factors and association with mortality
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Averbuch, Diana, Tridello, Gloria, Hoek, Jennifer, Mikulska, Malgorzata, Pabst, Thomas, Yanez San Segundo, Lucrecia, Akan, Hamdi, Ozcelik, Tulay, Donnini, Irene, Klyasova, Galina, Sousa, Aida Botelho, Zuckerman, Tsila, Wu, Depei, Tecchio, Cristina, La Camara, Rafael, Sahika Zeynep Aki, Ljungman, Per, Gulbas, Zafer, Nicolas, Emmanuelle, Calore, Elisabetta, Perruccio, Katia, Ram, Ron, Annaloro, Claudio, Martino, Rodrigo, Avni, Batia, Shaw, Peter, Gil, Lidia, Iacobelli, Simona, Styczynski, Jan, Engelhard, Dan, and Cesaro, Simone
10. Bortezomib(Velcade (R))-Thalidomide-Dexamethasone (VTD) Is Superior to Thalidomide-Dexamethasone (TD) In Patients with Multiple Myeloma (MM) Progressing or Relapsing After Autologous Transplantation
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Garderet, Laurent, Iacobelli, Simona, Moreau, Philippe, Dib, Mamoun, Caillot, Denis, Niederwieser, Dietger W., Masszi, Tamas, Fontan, Jean, Michallet, Mauricette, Gratwohl, Alois, Milone, Giuseppe, Doyen, Chantal, Pegourie, Brigitte, Roman Hajek, Casassus, Philippe, Kolb, Brigitte, Chaleteix, Carine, Hertenstein, Bernd, Onida, Francesco, Ludwig, Heinz, Vekemans, Marie Christiane, Ketterer, Nicolas, Daguenel, Anne, Gorin, Norbert C., Harousseau, Jean Luc, Witte, Theo, Morris, Curly, and Gahrton, Gosta
11. External validation of scores predicting non-relapse mortality in patients with myelodysplastic syndrome: a study from MDS subcommittee of the CMWP EBMT
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Carre, Martin, Porcher, Raphael, Finke, Juergen, Ehninger, Gerhard, Koster, Linda, Beelen, Dietrich, Ganser, Arnold, Volin, Liisa, Lozano, Sara, Sengeloev, Henrik, Michallet, Mauricette, Tischer, Johanna, Olavarria, Eduardo, Pascual Cascon, Maria Jesus, Iacobelli, Simona, Koc, Yener, Jindra, Pavel, Mutlu Arat, Witte, Theo, Kroeger, Nicolaus, and Robin, Marie
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Medizin
12. Impact of T-cell depletion strategies on outcomes following hematopoietic stem cell transplantation for idiopathic aplastic anemia: A study on behalf of the European blood and marrow transplant severe aplastic anemia working party
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Samarasinghe, S, Clesham, K, Iacobelli, S, Sbianchi, G, Knol, C, Hamladji, R-M, Socié, G, Aljurf, M, Koh, M, Sengeloev, H, Dalle, J-H, Robinson, S, Van Lint, MT, Halkes, CJ, Beelen, D, Mufti, GJ, Snowden, J, Blaise, D, Peffault de Latour, R, Marsh, J, Dufour, C, Risitano, AM, Severe Aplastic Anaemia Working Party of the EBMT, Samarasinghe, Sujith, Clesham, Katherine, Iacobelli, Simona, Sbianchi, Giulia, Knol, Cora, Hamladji, Rose-Marie, Socié, Gerard, Aljurf, Mahmoud, Koh, Mickey, Sengeloev, Henrik, Dalle, Jean-Hugue, Robinson, Stephen, Van Lint, Maria Teresa, Halkes, Constantijn J. M., Beelen, Dietrich, Mufti, Ghulam J., Snowden, John, Blaise, Didier, de Latour, Regis Peffault, Marsh, Judith, Dufour, Carlo, and Risitano, Antonio M
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Adult ,Male ,medicine.medical_specialty ,Transplantation Conditioning ,Adolescent ,Anemia ,T-Lymphocytes ,medicine.medical_treatment ,Medizin ,Graft vs Host Disease ,Hematopoietic stem cell transplantation ,Lower risk ,Gastroenterology ,Lymphocyte Depletion ,Young Adult ,03 medical and health sciences ,0302 clinical medicine ,Internal medicine ,Living Donors ,medicine ,Humans ,Young adult ,Child ,Alemtuzumab ,Aged ,Antilymphocyte Serum ,Retrospective Studies ,Transplant Conditioning ,business.industry ,Hematopoietic Stem Cell Transplantation ,Immunization, Passive ,Anemia, Aplastic ,Infant ,Hematology ,Middle Aged ,Settore MED/15 ,medicine.disease ,Transplantation ,Settore MED/01 ,Treatment Outcome ,surgical procedures, operative ,Child, Preschool ,030220 oncology & carcinogenesis ,Female ,business ,030215 immunology ,medicine.drug - Abstract
We retrospectively analyzed the outcomes of 1837 adults and children with severe aplastic anemia (SAA) who underwent matched sibling donor (MSD) and matched unrelated donor (MUD) hemopoietic stem cell transplantation (HSCT) between 2000 and 2013. Patients were grouped by transplant conditioning containing either anti‐thymocyte globulin (ATG) (n = 1283), alemtuzumab (n = 261), or no serotherapy (NS) (n = 293). The risks of chronic GvHD were significantly reduced when ATG or alemtuzumab were compared with NS (P = .021 and .003, respectively). Acute GVHD was significantly reduced in favor of alemtuzumab compared with ATG (P = .012) and NS (P < .001). By multivariate analysis, when compared with ATG, alemtuzumab was associated with a lower risk of developing acute (OR 0.262; 95% CI 0.14‐0.47; P < .001) and chronic GVHD (HR 0.58; 95% CI 0.35‐0.94; P = .027). OS was significantly better in ATG and alemtuzumab patients compared with NS (P = .010 and .025). Our data shows inclusion of serotherapy in MSD and MUD HSCT for patients with SAA reduces chronic GVHD and provides a survival advantage over patients not receiving serotherapy. Notably, alemtuzumab reduced the risk of acute and chronic GvHD compared with ATG and indicates that alemtuzumab might be the serotherapy of choice for MSD and MUD transplants for SAA.
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- 2018
13. Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita
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Julián Sevilla, Paul Veys, E T Korthof, Régis Peffault de Latour, Dorine Bresters, Tracey A. O'Brien, Marco Zecca, Maura Faraci, Simona Iacobelli, Jean Hugues Dalle, Antonio M. Risitano, Luca Arcuri, Maurizio Miano, Francesca Fioredda, Ardeshir Ghavamzadeh, Reuven Or, Miguel Angel Diaz, Brune Mats, E V Skorobogatova, Josue de la Fuente, Cora Knol, Petr Sedlacek, Franca Fagioli, Cristina Díaz de Heredia, Ayami Yoshimi, Michael Maschan, Jakub Tolar, Owen P. Smith, Carlo Dufour, Mahmoud Aljurf, Maria Teresa Van Lint, Alain Fisher, Anja van Biezen, Fioredda, Francesca, Iacobelli, Simona, Korthof, Elisabeth T., Knol, Cora, van Biezen, Anja, Bresters, Dorine, Veys, Paul, Yoshimi, Ayami, Fagioli, Franca, Mats, Brune, Zecca, Marco, Faraci, Maura, Miano, Maurizio, Arcuri, Luca, Maschan, Michael, O'Brien, Tracey, Diaz, Miguel A., Sevilla, Julian, Smith, Owen, Peffault de Latour, Regi, de la Fuente, Josue, Or, Reuven, Van Lint, Maria T., Tolar, Jakub, Aljurf, Mahmoud, Fisher, Alain, Skorobogatova, Elena V., Diaz de Heredia, Cristina, Risitano, Antonio, Dalle, Jean-Hugue, Sedláček, Petr, Ghavamzadeh, Ardeshir, and Dufour, Carlo
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Adult ,Male ,Oncology ,medicine.medical_specialty ,Transplantation Conditioning ,Pulmonary Fibrosis ,haematopoietic stem cell transplantation ,Graft vs Host Disease ,Disease ,dyskeratosis congenita ,Settore MED/01 - Statistica Medica ,Young Adult ,bone marrow failure ,Hematology ,03 medical and health sciences ,0302 clinical medicine ,Internal medicine ,medicine ,Humans ,Bone Marrow Diseases ,business.industry ,Age Factors ,Hematopoietic Stem Cell Transplantation ,Bone marrow failure ,medicine.disease ,Survival Analysis ,Tissue Donors ,Transplantation ,Haematopoiesis ,Treatment Outcome ,surgical procedures, operative ,medicine.anatomical_structure ,030220 oncology & carcinogenesis ,Female ,Bone marrow ,Stem cell ,business ,Dyskeratosis congenita ,030215 immunology - Abstract
Dyskeratosis congenita (DC) is a genetic multisystem disorder with frequent involvement of the bone marrow. Haematopoietic stem cell transplantation (HSCT) is the only definitive cure to restore haematopoiesis, even though it cannot correct other organ dysfunctions. We collected data on the outcome of HSCT in the largest cohort of DC (n = 94) patients ever studied. Overall survival (OS) and event-free survival (EFS) at 3 years after HSCT were 66% and 62%, respectively. Multivariate analysis showed better outcomes in patients aged less than 20 years and in patients transplanted from a matched, rather than a mismatched, donor. OS and EFS curves tended to decline over time. Early lethal events were infections, whereas organ damage and secondary malignancies appeared afterwards, even a decade after HSCT. A non-myeloablative conditioning regimen appeared to be most advisable. Organ impairment present before HSCT seemed to favour the development of chronic graft-versus-host disease and T-B immune deficiency appeared to enhance pulmonary fibrosis. According to the present data, HSCT in DC is indicated in cases of progressive marrow failure, whereas in patients with pre-existing organ damage, this should be carefully evaluated. Further efforts to investigate treatment alternatives to HSCT should be encouraged.
- Published
- 2018
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