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Hematopoietic cell transplantation for idiopathic severe aplastic anemia and Fanconi anemia in children and adolescents

Authors
Shakila Khan, MD
Robert S Negrin, MD
Section Editor
Donald H Mahoney, Jr, MD
Deputy Editor
Jennifer S Tirnauer, MD

INTRODUCTION

Aplastic anemia can result from either inherited or acquired causes. The incidence is triphasic, with small peaks at two to five years (secondary to inherited disorders) and 20 to 25 years, although the majority of patients present beyond 55 to 60 years of age.

The use of hematopoietic cell transplantation in children with idiopathic severe aplastic anemia and Fanconi anemia will be reviewed here. The causes and clinical presentation of acquired and inherited aplastic anemia and the use of hematopoietic cell transplantation in children with Diamond-Blackfan anemia and the myelodysplastic syndromes are discussed separately. (See "Acquired aplastic anemia in children and adolescents" and "Inherited aplastic anemia in children and adolescents" and "Hematopoietic cell transplantation for Diamond-Blackfan anemia and the myelodysplastic syndromes in children and adolescents".)

The term "hematopoietic cell transplantation" (HCT) will be used throughout this topic as a general term to cover transplantation of progenitor cells from any source (eg, bone marrow, peripheral blood, cord blood). Otherwise, the source of such cells will be specified (eg, autologous peripheral blood progenitor cell transplantation). (See "Sources of hematopoietic stem cells".)

IDIOPATHIC SEVERE APLASTIC ANEMIA

Idiopathic severe aplastic anemia (SAA) is a rare but serious disorder in children. The criteria for severe aplastic anemia (SAA) are [1]:

A bone marrow biopsy showing less than 25 percent of normal cellularity, OR

                     

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