Format

Send to

Choose Destination
Biol Blood Marrow Transplant. 2016 Oct;22(10):1888-1892. doi: 10.1016/j.bbmt.2016.06.018. Epub 2016 Jun 21.

Preemptive Bone Marrow Transplantation and Event-Free Survival in Fanconi Anemia.

Author information

1
Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland.
2
Biostatistics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland.
3
Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland. Electronic address: alterb@mail.nih.gov.

Abstract

Fanconi anemia (FA) is a rare inherited bone marrow failure syndrome associated with high risks of severe bone marrow failure (BMF), acute myeloid leukemia (AML), and solid tumors (ST). Bone marrow transplantation (BMT) provides a theoretical cure for hematologic risks (BMF, AML), but it introduces uncertain risks of transplantation-related mortality (TRM) and carcinogenicity. We developed a mathematical (Markov) decision model to estimate event-free survival (EFS) conditional on age based on per-year cause-specific hazard rates. We assumed that preemptive (PE) BMT eliminates the risks of BMF and AML, but it may introduce independent risks of TRM or influence the trajectory to ST. Our model suggested that the expected mean EFS in FA is higher for PE-BMT at young ages, with minimal risk of TRM and with little carcinogenicity. PE-BMT in adults decreased expected EFS because of the greater competing risk of ST in adulthood. Estimates of EFS conditioned on attained age may be used in shared decision-making when clinicians must counsel patients using limited data. Our methods may be used to model early transplantation in other blood disorders for which hematopoietic stem cell transplantation mitigates some but not all of the risks.

KEYWORDS:

Decision analysis; Fanconi anemia; Markov model; Preemptive transplantation

PMID:
27345141
PMCID:
PMC5066303
DOI:
10.1016/j.bbmt.2016.06.018
[Indexed for MEDLINE]
Free PMC Article

Supplemental Content

Full text links

Icon for Elsevier Science Icon for PubMed Central
Loading ...
Support Center