Late effects screening guidelines after hematopoietic cell transplantation for inherited bone marrow failure syndromes: Consensus statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on late effects after pediatric HCT Journal Article


Authors: Dietz, A. C.; Savage, S. A.; Vlachos, A.; Mehta, P. A.; Bresters, D.; Tolar, J.; Bonfim, C.; Dalle, J. H.; de la Fuente, J.; Skinner, R.; Boulad, F.; Duncan, C. N.; Baker, K. S.; Pulsipher, M. A.; Lipton, J. M.; Wagner, J. E.; Alter, B. P.
Article Title: Late effects screening guidelines after hematopoietic cell transplantation for inherited bone marrow failure syndromes: Consensus statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on late effects after pediatric HCT
Abstract: Patients with inherited bone marrow failure syndromes (IBMFS), such as Fanconi anemia (FA), dyskeratosis congenita (DC), or Diamond Blackfan anemia (DBA), can have hematologic manifestations cured through hematopoietic cell transplantation (HCT). Subsequent late effects seen in these patients arise from a combination of the underlying disease, the pre-HCT therapy, and the HCT process. During the international consensus conference sponsored by the Pediatric Blood and Marrow Transplant Consortium on late effects screening and recommendations following allogeneic hematopoietic cell transplantation for immune deficiency and nonmalignant hematologic diseases held in Minneapolis, Minnesota in May 2016, a half-day session was focused specifically on the unmet needs for these patients with IBMFS. This multidisciplinary group of experts in rare diseases and transplantation late effects has already published on the state of the science in this area, along with discussion of an agenda for future research. This companion article outlines consensus disease-specific long-term follow-up screening guidelines for patients with IMBFS. © 2017 The American Society for Blood and Marrow Transplantation
Keywords: late effects; fanconi anemia; dyskeratosis congenita; inherited bone marrow failure syndromes; diamond blackfan anemia; pediatric allogeneic hematopoietic cell transplant
Journal Title: Biology of Blood and Marrow Transplantation
Volume: 23
Issue: 9
ISSN: 1083-8791
Publisher: Elsevier Inc.  
Date Published: 2017-09-01
Start Page: 1422
End Page: 1428
Language: English
DOI: 10.1016/j.bbmt.2017.05.022
PROVIDER: scopus
PMCID: PMC5565711
PUBMED: 28533057
DOI/URL:
Notes: Conference Paper -- Export Date: 1 November 2017 -- Source: Scopus
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  1. Farid Boulad
    329 Boulad