Table 4. Management of hematologic... | American Society of Hematology

Table 4.

Management of hematologic complications in the inherited bone marrow failure syndromes and responses.¹

Treatment	Fanconi anemia	Dyskeratosis congenita	Diamond-Blackfan anemia	Shwachman-Diamond syndrome	Severe congenital neutropenia	Amega	Thrombocytopenia absent radii
Abbreviations: Amega, amegakaryocytic thrombocytopenia; G-CSF, granulocyte colony-stimulating factor. Yes, proven role; —, used rarely, role uncertain; No, role not proven and may be detrimental.
¹Response rates and durations vary according to the disorder.
²Transplantation regimens and successes depend on the disorder and the donor type.
Androgens	Yes	Yes	—	—	No	Yes	No
Prednisone	—	—	Yes	—	No	—	No
G-CSF	Rare	Rare	No	Yes	Yes	—	No
Erythropoietin	Rare	Rare	—
Red cell transfusions	As needed	As needed	First year; as needed	Rare	No	As needed	—
Platelet transfusions	As needed	As needed	No	Rare	No	As needed	First year; as needed
Stem cell transplantation²	Yes	Yes	Yes	Yes	Yes	Yes	Yes
“Spontaneous” remission	Mosaicism	Rare	~25%	Pancreatic function may improve with age	No	No	Yes

Treatment	Fanconi anemia	Dyskeratosis congenita	Diamond-Blackfan anemia	Shwachman-Diamond syndrome	Severe congenital neutropenia	Amega	Thrombocytopenia absent radii
Abbreviations: Amega, amegakaryocytic thrombocytopenia; G-CSF, granulocyte colony-stimulating factor. Yes, proven role; —, used rarely, role uncertain; No, role not proven and may be detrimental.
¹Response rates and durations vary according to the disorder.
²Transplantation regimens and successes depend on the disorder and the donor type.
Androgens	Yes	Yes	—	—	No	Yes	No
Prednisone	—	—	Yes	—	No	—	No
G-CSF	Rare	Rare	No	Yes	Yes	—	No
Erythropoietin	Rare	Rare	—
Red cell transfusions	As needed	As needed	First year; as needed	Rare	No	As needed	—
Platelet transfusions	As needed	As needed	No	Rare	No	As needed	First year; as needed
Stem cell transplantation²	Yes	Yes	Yes	Yes	Yes	Yes	Yes
“Spontaneous” remission	Mosaicism	Rare	~25%	Pancreatic function may improve with age	No	No	Yes