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.

• 影响指数: 5.609
• 发表时间: Sep 2017
• 来源期刊: Biol Blood Marrow Transplant
• DOI: 10.1016/j.bbmt.2017.05.022
• PMID: 28533057
• 文章类型: Journal Article
• 中科院分区: Q2
• 方向: 医学
• 作者列表: Dietz AC,Savage SA,Vlachos A,Mehta PA,Bresters D,Tolar J,Bonfim C,Dalle JH,de la Fuente J,Skinner R,Boulad F,Duncan CN,Baker KS,Pulsipher MA,Lipton JM,Wagner JE,Alter BP

关键词: Diamond Blackfan anemia; Dyskeratosis congenita; Fanconi anemia; Inherited bone marrow failure syndromes; Late effects; Pediatric allogeneic hematopoietic cell transplant

Mesh: 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

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.