What is the treatment of choice for a young patient with severe or very severe aplastic anemia (SAA or VSAA)?

Updated: Jan 29, 2021
  • Author: Sameer Bakhshi, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Human leukocyte antigen (HLA)-matched sibling-donor HCT is the treatment of choice for a young patient with severe or very severe aplastic anemia (SAA or VSAA, respectively), being generally accepted for patients younger than 50 years. [5] Persons undergoing this procedure do not require irradiation-based conditioning regimens. [5]

A study of 692 German patients with SAA who received transplants from HLA-matched siblings found that in patients younger than 20 years, rates of chronic graft versus host disease (GVHD) and overall mortality were higher after transplantation of peripheral blood progenitor cell (PBPC) grafts than after bone marrow transplants. [73] A multinational study of patients with SAA who received HCT from an HLA-matched sibling donor concluded that although bone marrow should definitely be the preferred graft source for these patients, PBPCs may be an acceptable alternative in countries with limited resources where patients present later in their disease course and risks of graft failure and infective complications are high. [74]


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