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

Andrew C. Dietz, Sharon A. Savage, Adrianna Vlachos, Parinda A. Mehta, Dorine Bresters, Jakub Tolar, Carmem Bonfim, Jean Hugues Dalle, Josu de la Fuente, Roderick Skinner, Farid Boulad, Christine N. Duncan, K. Scott Baker, Michael A. Pulsipher, Jeffrey M. Lipton, John E. Wagner, Blanche P. Alter

Research output: Contribution to journalArticlepeer-review

22 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)1422-1428
Number of pages7
JournalBiology of Blood and Marrow Transplantation
Volume23
Issue number9
DOIs
StatePublished - Sep 2017

Bibliographical note

Funding Information:
Financial disclosure: This work was supported in part by grants from the National Institutes of Health (1R13CA159788-01, to M.A.P. and K.S.B.; U01HL069254, to M.A.P.; R01 CA078938, to K.S.B.; and 5R01HL079571, to J.M.L. and A.V.), the Pediatric Blood and Marrow Transplant Consortium, and St. Baldrick's Foundation (U01HL069254, to M.A.P.). B.P.A. and S.A.S. are supported by the Intramural Research Program of the National Cancer Institute. The views expressed in this paper do not reflect the official policies of the Department of Health and Human Services, nor does mention of trade names, commercial practices, or organizations imply endorsement by the US Government. The content is solely the responsibility of the authors and does not necessarily represent the official views of those that provided funding.

Funding Information:
Financial disclosure: This work was supported in part by grants from the National Institutes of Health ( 1R13CA159788-01 , to M.A.P. and K.S.B.; U01HL069254 , to M.A.P.; R01 CA078938 , to K.S.B.; and 5R01HL079571 , to J.M.L. and A.V.), the Pediatric Blood and Marrow Transplant Consortium, and St. Baldrick's Foundation (U01HL069254, to M.A.P.). B.P.A. and S.A.S. are supported by the Intramural Research Program of the National Cancer Institute . The views expressed in this paper do not reflect the official policies of the Department of Health and Human Services, nor does mention of trade names, commercial practices, or organizations imply endorsement by the US Government. The content is solely the responsibility of the authors and does not necessarily represent the official views of those that provided funding. Conflict of interest statement: There are no conflicts of interest to report. Authorship statement: J.E.W. and B.P.A. contributed equally to this work.

Publisher Copyright:
© 2017 The American Society for Blood and Marrow Transplantation

Keywords

  • Diamond Blackfan anemia
  • Dyskeratosis congenita
  • Fanconi anemia
  • Inherited bone marrow failure syndromes
  • Late effects
  • Pediatric allogeneic hematopoietic cell transplant

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