9 results on '"van Biezen, Anja"'
Search Results
2. 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
3. Use of the quality management system 'JACIE' and outcome after hematopoietic stem cell transplantation
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Gratwohl, Alois, Brand, Roland, McGrath, Eoin, van Biezen, Anja, Sureda, Anna, Ljungman, Per, Baldomero, Helen, Chabannon, Christian, Apperley, Jane, Joint Accreditation Committee (JACIE), International Society for Cellular Therapy, European Group for Blood and Marrow Transplantatio, and European Leukemia Net
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Male ,Quality Control ,medicine.medical_specialty ,Outcome assessment (Medical care) ,Quality Assurance, Health Care ,medicine.medical_treatment ,Improved survival ,Hematopoietic stem cell transplantation ,Stem cells ,Accreditation ,Recurrence ,Risk Factors ,Outcome Assessment, Health Care ,medicine ,Overall survival ,Humans ,Mortality ,Intensive care medicine ,Retrospective Studies ,Patient Care Team ,business.industry ,Hazard ratio ,Hematopoietic Stem Cell Transplantation ,Hematology ,Articles ,Garantia de qualitat ,Quality assurance ,Transplantation ,Quality management system ,Cohort ,Emergency medicine ,Avaluació de resultats (Assistència mèdica) ,Female ,business ,Cèl·lules mare ,Total Quality Management - Abstract
Competent authorities, healthcare payers and hospitals devote increasing resources to quality management systems but scientific analyses searching for an impact of these systems on clinical outcome remain scarce. Earlier data indicated a stepwise improvement in outcome after allogeneic hematopoietic stem cell transplantation with each phase of the accreditation process for the quality management system “JACIE”. We therefore tested the hypothesis that working towards and achieving “JACIE” accreditation would accelerate improvement in outcome over calendar time. Overall mortality of the entire cohort of 107,904 patients who had a transplant (41,623 allogeneic, 39%; 66,281 autologous, 61%) between 1999 and 2006 decreased over the 14-year observation period by a factor of 0.63 per 10 years (hazard ratio: 0.63; 0.58–0.69). Considering “JACIE“-accredited centers as those with programs having achieved accreditation by November 2012, at the latest, this improvement was significantly faster in “JACIE”-accredited centers than in non-accredited centers (approximately 5.3% per year for 49,459 patients versus approximately 3.5% per year for 58,445 patients, respectively; hazard ratio: 0.83; 0.71–0.97). As a result, relapse-free survival (hazard ratio 0.85; 0.75–0.95) and overall survival (hazard ratio 0.86; 0.76–0.98) were significantly higher at 72 months for those patients transplanted in the 162 “JACIE“-accredited centers. No significant effects were observed after autologous transplants (hazard ratio 1.06; 0.99–1.13). Hence, working towards implementation of a quality management system triggers a dynamic process associated with a steeper reduction in mortality over the years and a significantly improved survival after allogeneic stem cell transplantation. Our data support the use of a quality management system for complex medical procedures.
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- 2014
4. Use Of a Quality Management System and Outcome After Hematopoietic Stem Cell Transplantation
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Jane F. Apperley, Ronald Brand, Christian Chabannon, Anna Sureda, van Biezen Anja, Helen Baldomero, Per Ljungman, Alois Gratwohl, and Eoin McGrath
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medicine.medical_specialty ,Allogeneic transplantation ,business.industry ,Incidence (epidemiology) ,medicine.medical_treatment ,Immunology ,Retrospective cohort study ,Cell Biology ,Hematology ,Hematopoietic stem cell transplantation ,Biochemistry ,Transplantation ,Emergency medicine ,Cohort ,Health care ,medicine ,business ,Accreditation - Abstract
Background Competent authorities, healthcare payers and hospitals devote increasing resources for quality management systems (QMS) and accreditation/certification of parts or all of their activities. Scientific analyses searching for an impact on clinical outcome remain scarce. Hematopoietic Stem Cell Transplantation (HSCT) involves complex processes and requires the collaboration of multiple categories of health care professionals. Despite major improvements over time, it remains associated with significant mortality and might serve as a role model for any QMS. The Joint Accreditation Committee of the International Society of Cellular Therapy Europe (ISCT) and the European Group for Blood and Marrow Transplantation (EBMT) – JACIE – has developed in collaboration with its US counterpart FACT standards that require a QMS. These principles apply to clinical management, cell collection and cell processing. Centers seeking accreditation are subject to a detailed document review, on-site inspection and auditing procedures. Earlier data indicated a stepwise improvement with each phase of the accreditation process (JCO 2011). Objective To test the hypothesis that working towards and achieving accreditation for the QMS “JACIE” would accelerate improvement in outcome over calendar time and, to quantify the potential impact on outcome. Design, setting and Patients A retrospective observational study of the previously published and well defined cohort of 107,904 patients treated with HSCT (41,623 allogeneic, 39%; 66,281 autologous, 61%) between 1999 and 2006, reported to the EBMT database and with substantially increased, complete follow-up information up to November 2012. A “JACIE+ “ center was defined as one which initiated the accreditation process and achieved accreditation at some point during the 14 year period, the latest by November 2012. Focus of the statistical approach was on the interaction between “JACIE+“ and calendar time on mortality reduction with adjustments for the key known risk factors. A completely different analysis framework has been chosen to test the hypothesis and to verify consistency between the two approaches. Main outcomes and measures: The primary outcome measure was reduction in overall mortality at 72 months over calendar time for 49,459 patients having received their transplant in a “JACIE+ “ center; 58,445 patients whose center never obtained accreditation served as baseline (“JACIE-”). Results Overall mortality of the entire cohort decreased over the 14 years observation period by a factor of 0.67 per 10 years (HR: 0.67; 0.62-0.73). This improvement was significantly faster in “JACIE+ “ (approx. 5% per year; HR per 10 years: HR: 0.62) than in “JACIE- “ (approx. 3% per year; HR per 10 years: HR: 0.73) centers. This difference in speed of mortality reduction was quantified by a HR of 0.85 (0.73-0.99) and resulted in a marked difference in outcome between the “JACIE+” and “JACIE-” centers: adjusted non relapse mortality (HR 0.93; 0.83-1.04) and relapse incidence (HR 0.94; 0.88-0.99) were lower, while relapse free (HR 0.94; 0.88-0.99) and overall survival (HR 0.91; 0.85-0.97) were significantly higher at 72 months after allogeneic transplantation for those patients transplanted in the 162 “JACIE+ “ centers. No effects of “JACIE” accreditation were observed after autologous HSCT, not on speed of mortality reduction nor on overall survival (HR 1.06; 0.99-1.13). Conclusions and relevance Working towards and implementation of a QMS triggers a dynamic process which is associated with a steeper reduction in mortality over the years and a significantly improved survival after allogeneic HSCT. Our data support the use of a QMS for allogeneic HSCT as well as for other forms of complex medical procedures. Disclosures: No relevant conflicts of interest to declare.
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- 2013
5. Beneficial role of CD8+T-cell reconstitution after HLA-haploidentical stem cell transplantation for high-risk acute leukaemias
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Bondanza, A., Ruggeri, L., Noviello, M., Eikema, D.J., Bonini, C., Chabannon, C., Werf, S. van der, Biezen, A. van, Wreede, L.C. de, Crucitti, L., Vago, L., Merluzzi, M., Massei, M.S., Veelken, H., Koc, Y., Bader, P., Gruhn, B., Locatelli, F., Ciceri, F., Toubert, A., Velardi, A., Bernardo, M.E., Dazzi, F., Ellard, R., Fleischhauer, K., Greco, R., Hudecek, M., Kohl, U., Kuball, J., Malard, F., Pedrazzoli, P., Rocha, V., Ruggeri, A., Urbano-Ispizua, A., Wang, J.F., Wieten, L., EBMT Cell Therapy Immunobiol Worki, Bondanza, Attilio, Ruggeri, Loredana, Noviello, Maddalena, Eikema, Dirk-Jan, Bonini, Chiara, Chabannon, Christian, van der Werf, Steffie, van Biezen, Anja, de Wreede, Liesbeth C., Crucitti, Lara, Vago, Luca, Merluzzi, Mara, Massei, Maria Speranza, Veelken, Hendrik, Koc, Yener, Bader, Peter, Gruhn, Bernd, Locatelli, Franco, Ciceri, Fabio, Toubert, Antoine, Velardi, Andrea, MUMC+: DA TI Laboratorium (9), RS: GROW - R3 - Innovative Cancer Diagnostics & Therapy, and MUMC+: DA TI Staf (9)
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Male ,Oncology ,BLOOD ,COUNT ,Medizin ,CHILDREN ,CD8-Positive T-Lymphocytes ,DISEASE ,0302 clinical medicine ,Risk Factors ,hemic and lymphatic diseases ,Cytotoxic T cell ,Registries ,Child ,INDEX ,Leukemia ,Hematology ,Middle Aged ,RECOVERY ,Haematopoiesis ,surgical procedures, operative ,medicine.anatomical_structure ,Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA ,Transplantation ,Child, Preschool ,030220 oncology & carcinogenesis ,HSCT ,Female ,Stem cell ,Adult ,medicine.medical_specialty ,Adolescent ,T cell ,Human leukocyte antigen ,Young Adult ,03 medical and health sciences ,CD8-positive T-lymphocytes ,haematopoietic stem cell transplantation ,risk factors ,Internal medicine ,SCORE ,medicine ,Humans ,IMMUNOTHERAPY ,Aged ,business.industry ,ADULTS ,BONE-MARROW-TRANSPLANTATION ,Transplantation, Haploidentical ,business ,CD8 ,Stem Cell Transplantation ,030215 immunology ,RESPONSES - Abstract
HLA-haploidentical haematopoietic stem cell transplantation (haplo-HSCT) is increasingly offered to patients with high-risk acute leukaemia. Unfortunately, haplo-HSCT is followed by a delayed immunoreconstitution. The aim of this EBMT registry study was to explore the clinical impact of lymphocyte subset counts after haplo-HSCT. We considered 144 leukaemic patients transplanted in the period 2001-2012. Pre-transplantation clinical variables and differential immune-cell counts (CD3, CD4, CD8 T cells, NK and B cells) measured before day 100 were evaluated for their capacity to predict overall survival, relapse mortality or non-relapse mortality (NRM). Negative prognostic factors for overall survival were advanced disease state at transplantation, host age and CMV seropositivity. Higher CD3, CD4 and CD8 counts were associated with a better overall survival and a lower NRM. Strikingly, when tested in multivariable analysis, higher CD3 and CD8 counts were still significantly associated with a lower NRM. These results indicate that an accelerated T-cell reconstitution correlates with less transplantation mortality, likely due to the protective role of T cells against viral infections. This observation suggests that CD8+ T-cell counts should be investigated as surrogate biomarkers of outcome in prospective haplo-HSCT trials.
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- 2019
6. 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.
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- 2018
7. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT
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Persis Amrolia, Rachael Hough, Sujith Samarasinghe, Judith C. W. Marsh, Mark Velangi, Peter Bader, Ajay Vora, Brenda Gibson, Antonio M. Risitano, Austin G. Kulasekararaj, Jakob Passweg, Neha Bhatnagar, Anna Maria Ewins, Alicia Rovó, Roderick Skinner, Régis Peffault de Latour, Mahmoud Aljurf, Andrea Bacigalupo, Marta Pillon, Elisa Carraro, Josu de la Fuente, Colin G. Steward, Britta Höchsmann, Hubert Schrezenmeier, Anja van Biezen, André Tichelli, Carlo Dufour, Rob Wynn, Paul Veys, Gérard Socié, Dufour, Carlo, Veys, Paul, Carraro, Elisa, Bhatnagar, Neha, Pillon, Marta, Wynn, Rob, Gibson, Brenda, Vora, Ajay J., Steward, Colin G., Ewins, Anna M., Hough, Rachael E., de la Fuente, Josu, Velangi, Mark, Amrolia, Persis J., Skinner, Roderick, Bacigalupo, Andrea, Risitano, ANTONIO MARIA, Socie, Gerard, Peffault de Latour, Regi, Passweg, Jakob, Rovo, Alicia, Tichelli, André, Schrezenmeier, Hubert, Hochsmann, Britta, Bader, Peter, van Biezen, Anja, Aljurf, Mahmoud D., Kulasekararaj, Austin, Marsh, Judith C., and Samarasinghe, Sujith
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Male ,Pediatrics ,Blood transfusion ,Adenoviridae Infection ,medicine.medical_treatment ,Adenoviridae Infections ,T-Lymphocytes ,Graft vs Host Disease ,Kaplan-Meier Estimate ,Immunosuppressive Agent ,Postoperative Complications ,Retrospective Studie ,hemic and lymphatic diseases ,Living Donors ,Young adult ,Child ,610 Medicine & health ,Bone Marrow Transplantation ,Herpesviridae Infection ,Anemia, Aplastic ,paediatric aplastic anaemia ,Hematology ,Herpesviridae Infections ,aplastic anaemia ,Survival Rate ,Treatment Outcome ,surgical procedures, operative ,Child, Preschool ,Histocompatibility ,Cohort ,Cyclosporine ,Female ,Case-Control Studie ,Immunosuppressive Agents ,Human ,Adult ,Living Donor ,medicine.medical_specialty ,Sibling ,Adolescent ,Disease-Free Survival ,Follow-Up Studie ,Young Adult ,medicine ,Humans ,Blood Transfusion ,Survival rate ,Antilymphocyte Serum ,Retrospective Studies ,Peripheral Blood Stem Cell Transplantation ,business.industry ,Siblings ,Case-control study ,Infant ,Retrospective cohort study ,Length of Stay ,Transplantation ,T-Lymphocyte ,Case-Control Studies ,Quality of Life ,Virus Activation ,Postoperative Complication ,Primary Graft Dysfunction ,business ,Follow-Up Studies ,transplantation - Abstract
We explored the feasibility of unrelated donor haematopoietic stem cell transplant (HSCT) upfront without prior immunosuppressive therapy (IST) in paediatric idiopathic severe aplastic anaemia (SAA). This cohort was then compared to matched historical controls who had undergone first-line therapy with a matched sibling/family donor (MSD) HSCT (n = 87) or IST with horse antithymocyte globulin and ciclosporin (n = 58) or second-line therapy with unrelated donor HSCT post-failed IST (n = 24). The 2-year overall survival in the upfront cohort was 96 ± 4% compared to 91 ± 3% in the MSD controls (P = 0·30) and 94 ± 3% in the IST controls (P = 0·68) and 74 ± 9% in the unrelated donor HSCT post-IST failure controls (P = 0·02).The 2-year event-free survival in the upfront cohort was 92 ± 5% compared to 87 ± 4% in MSD controls (P = 0·37), 40 ± 7% in IST controls (P = 0·0001) and 74 ± 9% in the unrelated donor HSCT post-IST failure controls (n = 24) (P = 0·02). Outcomes for upfront-unrelated donor HSCT in paediatric idiopathic SAA were similar to MSD HSCT and superior to IST and unrelated donor HSCT post-IST failure. Front-line therapy with matched unrelated donor HSCT is a novel treatment approach and could be considered as first-line therapy in selected paediatric patients who lack a MSD. © 2015 John Wiley & Sons Ltd.
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- 2015
8. Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant
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Mahmoud Aljurf, Christina Peters, Judith C. W. Marsh, Ayad Ahmed Hussein, Mouhab Ayas, Antonio M. Risitano, Régis Peffault de Latour, Anja van Biezen, Alicia Rovó, André Tichelli, Sujith Samarasinghe, Carlo Dufour, Jakob Passweg, Austin G. Kulasekararaj, Marta Pillon, Gérard Socié, Britta Hocshmann, Hubert Schrezenmeier, Andrea Bacigalupo, Elisa Carraro, Rosi Oneto, Dufour, Carlo, Pillon, Marta, Sociè, Gerard, Rovò, Alicia, Carraro, Elisa, Bacigalupo, Andrea, Oneto, Rosi, Passweg, Jakob, Risitano, ANTONIO MARIA, Tichelli, Andrè, Peffault de Latour, Regi, Schrezenmeier, Hubert, Hocshmann, Britta, Peters, Christina, Kulasekararaj, Austin, Van Biezen, Anja, Samarasinghe, Sujith, Hussein, Ayad Ahmed, Ayas, Mouhab, Aljurf, Mahmoud, and Marsh, Judith
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Graft Rejection ,Male ,Databases, Factual ,medicine.medical_treatment ,Graft vs Host Disease ,Aplastic anaemia ,Disease ,Hematopoietic stem cell transplantation ,Allograft ,immune system diseases ,hemic and lymphatic diseases ,Child ,610 Medicine & health ,Children ,Marrow transplantation ,Haematopoietic stem cell transplantation ,Aplastic ,Hematopoietic Stem Cell Transplantation ,Anemia, Aplastic ,Anemia ,Immunosuppression ,Hematology ,Allografts ,Europe ,Survival Rate ,surgical procedures, operative ,Child, Preschool ,Female ,Human ,medicine.medical_specialty ,Bone marrow transplant ,chemical and pharmacologic phenomena ,Disease-Free Survival ,Databases ,Internal medicine ,medicine ,Overall survival ,Humans ,Preschool ,Survival rate ,Factual ,Immunosuppression Therapy ,business.industry ,Infant, Newborn ,Infant ,Newborn ,Surgery ,Transplantation ,Settore MED/15 - MALATTIE DEL SANGUE ,business - Abstract
This study analysed the outcome of 563 Aplastic Anaemia (AA) children aged 0-12 years reported to the Severe Aplastic Anaemia Working Party database of the European Society for Blood and Marrow Transplantation, according to treatment received. Overall survival (OS) after upfront human leucocyte antigen-matched family donor (MFD) haematopoietic stem cell transplantation (HSCT) or immunosuppressive treatment (IST) was 91% vs. 87% (P 0·18). Event-free survival (EFS) after upfront MFD HSCT or IST was 87% vs. 33% (P 0·001). Ninety-one of 167 patients (55%) failed front-line IST and underwent rescue HSCT. The OS of this rescue group was 83% compared with 91% for upfront MFD HSCT patients and 97% for those who did not fail IST up-front (P 0·017). Rejection was 2% for MFD HSCT and HSCT post-IST failure (P 0·73). Acute graft-versus-host disease (GVHD) grade II-IV was 8% in MFD graft vs. 25% for HSCT post-IST failure (P < 0·0001). Chronic GVHD was 6% in MFD HSCT vs. 20% in HSCT post-IST failure (P < 0·0001). MFD HSCT is an excellent therapy for children with AA. IST has a high failure rate, but remains a reasonable first-line choice if MFD HSCT is not available because high OS enables access to HSCT, which is a very good rescue option.
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- 2015
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9. Stem cell transplantation in severe congenital neutropenia: An analysis from the European Society for Blood and Marrow Transplantation
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Fioredda, F., Iacobelli, S., Biezen, A. van, Gaspar, B., Ancliff, P., Donadieu, J., Aljurf, M., Peters, C., Calvillo, M., Matthes-Martin, S., Morreale, G., van't Veer-Tazelaar, N., Wreede, L. de, Seraihy, A. al, Yesilipek, A., Fischer, A., Bierings, M., Ozturk, G., Smith, O., Veys, P., Ljungman, P., Latour, R.P. de, Codina, J.S.D., Or, R., Ganser, A., Afanasyev, B., Wynn, R., Kalwak, K., Marsh, J., Dufour, C., Pediat Dis Working Parties Europea, Fioredda, Francesca, Iacobelli, Simona, Van Biezen, Anja, Gaspar, Bobby, and Risitano, ANTONIO MARIA
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Male ,Graft Rejection ,medicine.medical_treatment ,Graft vs Host Disease ,Hematopoietic stem cell transplantation ,Biochemistry ,Allograft ,Retrospective Studie ,Cumulative incidence ,Non-U.S. Gov't ,Child ,Societies, Medical ,Incidence (epidemiology) ,Research Support, Non-U.S. Gov't ,Histocompatibility Testing ,Incidence ,Medicine (all) ,Hematopoietic Stem Cell Transplantation ,Hematology ,Allografts ,Clinical Trial ,Multicenter Study ,Europe ,surgical procedures, operative ,Child, Preschool ,Acute Disease ,Cyclosporine ,Female ,Unrelated Donors ,Kostmann syndrome ,Human ,Adult ,medicine.medical_specialty ,Neutropenia ,Adolescent ,Immunology ,Research Support ,Settore MED/01 - Statistica Medica ,Middle East ,Internal medicine ,Medical ,medicine ,Journal Article ,Humans ,Aplastic anemia ,Congenital Neutropenia ,Preschool ,Retrospective Studies ,business.industry ,Unrelated Donor ,Cell Biology ,medicine.disease ,Surgery ,Transplantation ,Methotrexate ,business ,Societies - Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment of severe congenital neutropenia (SCN), but data on outcome are scarce. We report on the outcome of 136 SCN patients who underwent HSCT between 1990 and 2012 in European and Middle East centers. The 3-year overall survival (OS) was 82%, and transplant-related mortality (TRM) was 17%. In multivariate analysis, transplants performed under the age of 10 years, in recent years, and from HLA-matched related or unrelated donors were associated with a significantly better OS. Frequency of graft failure was 10%. Cumulative incidence (day +90) of acute graft-versus-host disease (GVHD) grade 2-4 was 21%. In multivariate analysis, HLA-matched related donor and prophylaxis with cyclosporine A and methotrexate were associated with lower occurrence of acute GVHD. Cumulative incidence (1 year) of chronic GVHD was 20%. No secondary malignancies occurred after a median follow-up of 4.6 years. These data show that the outcome of HSCT for SCN from HLA-matched donors, performed in recent years, in patients younger than 10 years is acceptable. Nevertheless, given the TRM, a careful selection of HSCT candidates should be undertaken.
- Published
- 2015
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