دورية أكاديمية
أعرض في EDS

Four decades of stem cell transplantation for Fanconi anaemia in the Netherlands.

التفاصيل البيبلوغرافية
العنوان: Four decades of stem cell transplantation for Fanconi anaemia in the Netherlands.
المؤلفون: Smetsers SE; Department of Paediatric Haematology and Stem Cell Transplantation, University Medical Centre Utrecht, Utrecht, The Netherlands., Smiers FJ; Department of Paediatric Haematology and Stem Cell Transplantation, Leiden University Medical Centre, Leiden, The Netherlands., Bresters D; Department of Paediatric Haematology and Stem Cell Transplantation, Leiden University Medical Centre, Leiden, The Netherlands., Sonnevelt MC; Department of Paediatric Haematology and Stem Cell Transplantation, University Medical Centre Utrecht, Utrecht, The Netherlands., Bierings MB; Department of Paediatric Haematology and Stem Cell Transplantation, University Medical Centre Utrecht, Utrecht, The Netherlands.; Dutch Childhood Oncology Group, Fanconi Anaemia Guideline Chair, The Hague, The Netherlands.
المصدر: British journal of haematology [Br J Haematol] 2016 Sep; Vol. 174 (6), pp. 952-61. Date of Electronic Publication: 2016 Jul 29.
نوع المنشور: Journal Article; Research Support, Non-U.S. Gov't
اللغة: English
بيانات الدورية: Publisher: Wiley-Blackwell Country of Publication: England NLM ID: 0372544 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2141 (Electronic) Linking ISSN: 00071048 NLM ISO Abbreviation: Br J Haematol Subsets: MEDLINE
أسماء مطبوعة: Publication: Oxford : Wiley-Blackwell
Original Publication: Oxford : Blackwell Scientific Publications
مواضيع طبية MeSH: Hematopoietic Stem Cell Transplantation*/adverse effects , Hematopoietic Stem Cell Transplantation*/methods, Fanconi Anemia/*epidemiology , Fanconi Anemia/*therapy, Adolescent ; Adult ; Child ; Child, Preschool ; Fanconi Anemia/diagnosis ; Fanconi Anemia/mortality ; Female ; Graft Rejection ; Graft Survival ; Graft vs Host Disease/diagnosis ; Graft vs Host Disease/etiology ; Humans ; Kaplan-Meier Estimate ; Male ; Mortality ; Netherlands/epidemiology ; Odds Ratio ; Retrospective Studies ; Transplantation Conditioning/methods ; Treatment Outcome ; Young Adult
مستخلص: This article presents the haematopoietic stem cell transplantation (SCT) results of the complete Dutch Fanconi anaemia (FA) patient cohort. Sixty-eight Dutch FA patients have been transplanted since 1972. In total, 63 (93%) patients engrafted, 54 after first SCT and 9 after second SCT. Fludarabine (FLU)-based conditioning was associated with decreased graft failure (odds ratio 0·21, P = 0·01), decreased early mortality (hazard ratio 0·25, P = 0·01) and improved 5-year overall survival (FLU 87·8% [standard error (SE) 5·1%] versus non-FLU 59·3% [SE 9·5%], P = 0·01). Late mortality was mainly caused by squamous cell carcinoma. Twenty-two patients were treated with the current Dutch FA conditioning regimen (FLU 150 mg/m(2) and cyclophosphamide 30 mg/kg ± anti-thymocyte globulin - no irradiation). Stem cell donors were matched related (n = 8) or alternative donors (n = 14). Stable engraftment after first SCT was achieved in 19 (86%) patients. At a median follow-up of 3·9 years 20 (91%) patients are alive. Our study provides a unique overview of a nation-wide SCT cohort illustrating the major improvements in treatment regimen and patient outcome in recent years. It shows that a non-irradiation and busulfan-free conditioning regimen can be used successfully, also in alternative donor SCT. Furthermore, it underlines the importance of late cancer screening and comprehensive care for this complex disorder.
(© 2016 John Wiley & Sons Ltd.)
فهرسة مساهمة: Keywords: Fanconi anaemia; bone marrow failure; chemosensitivity; leukaemia; stem cell transplantation
تواريخ الأحداث: Date Created: 20160730 Date Completed: 20170515 Latest Revision: 20180208
رمز التحديث: 20240628
DOI: 10.1111/bjh.14165
PMID: 27470218
قاعدة البيانات: MEDLINE
الوصف
تدمد:1365-2141
DOI:10.1111/bjh.14165