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

Summary 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 mortalit... Mehr ...

Verfasser: Smetsers, Stephanie E.
Smiers, Frans J.
Bresters, Dorine
Sonnevelt, Martine C.
Bierings, Marc B.
Dokumenttyp: Artikel
Erscheinungsdatum: 2016
Reihe/Periodikum: British Journal of Haematology ; volume 174, issue 6, page 952-961 ; ISSN 0007-1048 1365-2141
Verlag/Hrsg.: Wiley
Sprache: Englisch
Permalink: https://search.fid-benelux.de/Record/base-27629590
Datenquelle: BASE; Originalkatalog
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Link(s) : http://dx.doi.org/10.1111/bjh.14165

Summary 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.