TY - JOUR
T1 - Late Effects Screening Guidelines after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes
T2 - Consensus Statement From the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects After Pediatric HCT
AU - Dietz, Andrew C.
AU - Savage, Sharon A.
AU - Vlachos, Adrianna
AU - Mehta, Parinda A.
AU - Bresters, Dorine
AU - Tolar, Jakub
AU - Bonfim, Carmem
AU - Dalle, Jean Hugues
AU - de la Fuente, Josu
AU - Skinner, Roderick
AU - Boulad, Farid
AU - Duncan, Christine N.
AU - Baker, K. Scott
AU - Pulsipher, Michael A.
AU - Lipton, Jeffrey M.
AU - Wagner, John E.
AU - Alter, Blanche P.
N1 - Publisher Copyright:
© 2017 The American Society for Blood and Marrow Transplantation
PY - 2017/9
Y1 - 2017/9
N2 - 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.
AB - 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.
KW - Diamond Blackfan anemia
KW - Dyskeratosis congenita
KW - Fanconi anemia
KW - Inherited bone marrow failure syndromes
KW - Late effects
KW - Pediatric allogeneic hematopoietic cell transplant
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U2 - 10.1016/j.bbmt.2017.05.022
DO - 10.1016/j.bbmt.2017.05.022
M3 - Article
C2 - 28533057
AN - SCOPUS:85021301439
SN - 1083-8791
VL - 23
SP - 1422
EP - 1428
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 9
ER -