Androgen therapy in Fanconi anemia

A retrospective analysis of 30 years in Germany

Laura Paustian, Mwe Mwe Chao, Helmut Hanenberg, Detlev Schindler, Heidemarie Neitzel, Christian Peter Kratz, Wolfram Ebell

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

A substantial number of individuals with Fanconi anemia (FA) develop bone marrow failure and are treated with androgen therapy in order to increase blood counts. The authors retrospectively identified 70 patients who received androgen therapy any time between July 1976 and September 2014. Among these patients, 37 had medical records for analysis. Twenty-five of the 37 (68%) patients had response in hemoglobin level (n = 25), platelet count (n = 21), and/or absolute neutrophil count (n = 13). The median rise in hemoglobin was 6.5 mg/dL, platelet count 70,000/mm3, and absolute neutrophil count (ANC) 1530/μL. The majority of patients (n = 22) had a response in 2 or more blood parameters. Reasons for discontinuation of therapy included development of cytogenetic aberrations (n = 9), lack of response (n = 7), hepatic adenoma (n = 6), progression to myelodysplastic syndrome/acute myeloid leukemia (n = 3), stabilization of blood parameters (n = 3), resolution of cytopenia secondary to mosaicism (n = 1), virilization (n = 1), development of anogenital carcinoma (n = 1), inaccessibility of medication (n = 1), and unknown (n = 1). Four patients at last follow-up remain on androgen therapy. These results highlight that androgen therapy can significantly improve blood counts for many FA patients, but progression of underlying bone marrow disease and development of liver adenomas requires careful monitoring.

Original languageEnglish (US)
Pages (from-to)1-8
Number of pages8
JournalPediatric Hematology and Oncology
DOIs
StateAccepted/In press - Jan 18 2016
Externally publishedYes

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Fanconi Anemia
Androgens
Germany
Platelet Count
Adenoma
Hemoglobins
Neutrophils
Therapeutics
Bone Marrow Diseases
Virilism
Mosaicism
Liver
Bone Development
Myelodysplastic Syndromes
Acute Myeloid Leukemia
Chromosome Aberrations
Medical Records
Bone Marrow
Carcinoma

Keywords

  • Androgen therapy
  • bone marrow failure
  • Fanconi anemia

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

Paustian, L., Chao, M. M., Hanenberg, H., Schindler, D., Neitzel, H., Kratz, C. P., & Ebell, W. (Accepted/In press). Androgen therapy in Fanconi anemia: A retrospective analysis of 30 years in Germany. Pediatric Hematology and Oncology, 1-8. https://doi.org/10.3109/08880018.2015.1129567

Androgen therapy in Fanconi anemia : A retrospective analysis of 30 years in Germany. / Paustian, Laura; Chao, Mwe Mwe; Hanenberg, Helmut; Schindler, Detlev; Neitzel, Heidemarie; Kratz, Christian Peter; Ebell, Wolfram.

In: Pediatric Hematology and Oncology, 18.01.2016, p. 1-8.

Research output: Contribution to journalArticle

Paustian, Laura ; Chao, Mwe Mwe ; Hanenberg, Helmut ; Schindler, Detlev ; Neitzel, Heidemarie ; Kratz, Christian Peter ; Ebell, Wolfram. / Androgen therapy in Fanconi anemia : A retrospective analysis of 30 years in Germany. In: Pediatric Hematology and Oncology. 2016 ; pp. 1-8.
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