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.

• Authors: 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
• Journal: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (Biol Blood Marrow Transplant) Vol. 23 Issue 9 Pg. 1422-1428 (Sep 2017) ISSN: 1523-6536 [Electronic] United States
• PMID: 28533057 (Publication Type: Journal Article, Practice Guideline, Review)
• Copyright: Copyright © 2017 The American Society for Blood and Marrow Transplantation. All rights reserved.

Topics

• Anemia, Aplastic (diagnosis, immunology, pathology, therapy)
• Anemia, Diamond-Blackfan (diagnosis, immunology, mortality, therapy)
• Bone Marrow Diseases (diagnosis, immunology, pathology, therapy)
• Bone Marrow Failure Disorders
• Child
• Consensus
• Consensus Development Conferences as Topic
• Dyskeratosis Congenita (diagnosis, immunology, mortality, therapy)
• Fanconi Anemia (diagnosis, immunology, mortality, therapy)
• Hematopoietic Stem Cell Transplantation
• Hemoglobinuria, Paroxysmal (diagnosis, immunology, pathology, therapy)
• Humans
• International Cooperation
• Survival Analysis
• Transplantation, Homologous