Table 1. Androgen therapy for inherited... | American Society of Hematology

Table 1.

Androgen therapy for inherited bone marrow failure syndromes

Study	Reference	Patients, N	Disease	Androgen	Median age, years	Median duration of therapy, years	Response, %	Toxicity
Scheckenbach et al.	14	8	Fanconi anemia	Danazol	11	3	87	—
Rose et al.*	15	10	Fanconi anemia	Oxandrolone	9	2	70	Virilization, liver, MDS
Paustian et al.	6	37	Fanconi anemia	Various	8.8	4.2†	68	Virilization, MDS, hepatic adenoma
Ribeiro et al.	16	66	Fanconi anemia	Oxymetholone, danazol	10.5	1.5	78	Virilization, liver
Khincha et al.	8	16	Dyskeratosis congenita	Oxymetholone, fluoxymesterone, nandrolone decanoate	11	2.2	69	Bone fractures, virilization, splenic peliosis, liver
Townsley et al.*	7	27	Telomeropathy	Danazol	41	2	83	Liver, muscle

Study	Reference	Patients, N	Disease	Androgen	Median age, years	Median duration of therapy, years	Response, %	Toxicity
Scheckenbach et al.	14	8	Fanconi anemia	Danazol	11	3	87	—
Rose et al.*	15	10	Fanconi anemia	Oxandrolone	9	2	70	Virilization, liver, MDS
Paustian et al.	6	37	Fanconi anemia	Various	8.8	4.2†	68	Virilization, MDS, hepatic adenoma
Ribeiro et al.	16	66	Fanconi anemia	Oxymetholone, danazol	10.5	1.5	78	Virilization, liver
Khincha et al.	8	16	Dyskeratosis congenita	Oxymetholone, fluoxymesterone, nandrolone decanoate	11	2.2	69	Bone fractures, virilization, splenic peliosis, liver
Townsley et al.*	7	27	Telomeropathy	Danazol	41	2	83	Liver, muscle

MDS, myelodysplastic syndrome.

Prospective study.

For responders.