Work

A Prospective Trial of Minimal Intensity Conditioning with Fludarabine and Anti-CD52 Antibody Alone in Dyskeratosis Congenita

Year: 2014

Type: article

Abstract: Dyskeratosis congenita (DC) is an inherited bone marrow failure (BMF) syndrome classically presenting with a clinical triad of skin pigment abnormalities, nail dystrophy, and oral leukoplakia. Progressive BMF occurs in approximately 80% of patients by 30 years of age and is the primary cause of death, followed by pulmonary failure and malignancies. HCT for DC is limited by a high incidence of treatment-related mortality, due to the exacerbation of underlying multi-organ disease by alkylating agents and radiation in the conditioning regimen.

Source: Transplantation and Cellular Therapy

DOI: 10.1016/j.bbmt.2013.12.266

Authors: Leslie E. Lehmann, David A. Williams, Wendy B. London, Suneet Agarwal

Institutions: Boston Children's Hospital, Dana-Farber Cancer Institute

Language: English

Topic: Hematopoietic Stem Cell Transplantation

Subfield: Hematology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $3,650