1. 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
- Author
-
Roderick Skinner, Jean Hugues Dalle, Michael A. Pulsipher, Jeffrey M. Lipton, Carmem Bonfim, Dorine Bresters, Adrianna Vlachos, Jakub Tolar, Parinda A. Mehta, Sharon A. Savage, Andrew C. Dietz, Blanche P. Alter, John E. Wagner, Farid Boulad, Josu de la Fuente, Christine Duncan, and K. Scott Baker
- Subjects
medicine.medical_specialty ,Pediatrics ,Consensus ,hematopoietic cell transplant ,Consensus Development Conferences as Topic ,International Cooperation ,Hemoglobinuria, Paroxysmal ,Article ,Unmet needs ,Pediatric allogeneic ,03 medical and health sciences ,0302 clinical medicine ,Fanconi anemia ,hemic and lymphatic diseases ,medicine ,Humans ,Transplantation, Homologous ,Diamond Blackfan anemia ,Diamond–Blackfan anemia ,Intensive care medicine ,Child ,Bone Marrow Diseases ,Anemia, Diamond-Blackfan ,Transplantation ,Hematopoietic cell ,business.industry ,Late effects ,Hematopoietic Stem Cell Transplantation ,Anemia, Aplastic ,Hematology ,Bone Marrow Failure Disorders ,medicine.disease ,Survival Analysis ,Inherited bone marrow failure syndromes ,Bone transplantation ,030220 oncology & carcinogenesis ,Bone Marrow failure syndromes ,business ,Dyskeratosis congenita ,030215 immunology - 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.
- Published
- 2017