Is there a role for transplant in aplastic anemia?

Marcie Riches, MD, MS
Clinical Associate Professor and Director of Clinical Research and Data Quality, BMT in the Division of Hematology/Oncology at the University of North Carolina at Chapel Hill

This month, Marcie Riches, MD, MS, answers a question about stem cell transplantation in a patient who presents with aplastic anemia and PNH.


A 28-year-old patient presented with aplastic anemia (AA) (diagnosed at age 15) and paroxysmal nocturnal hemoglobinuria (PNH). A bone marrow biopsy showed 15 percent cellularity. He responded to cyclosporine and ATG but was lost to follow-up. When he came back about eight years ago, he was noted to have pancytopenia with hemolysis and a PNH clone was found, representing 12.4 percent of his total RBCs. He was prescribed eculizumab and though improvement was observed, blood counts did not normalize. His last bone marrow biopsy was normocellular. Is there a role for stem cell transplantation in this patient?


This is an interesting case. I have some questions that are relevant to my response regarding the role of stem cell transplantation.

As I understand from your case, this is a 28-year-old male who at 15 years old had AA (15% cellular bone marrow) treated with CSA and ATG. He was lost to follow-up for five years and resurfaced eight years ago with pancytopenia and a small PNH clone.

  1. How severe was his pancytopenia at re-presentation?
    A: 2010: WBC 2.6, HGB 8, Plts 55K, ANC 1.5; 2015: WBC 3.6, HGB ~10, Plts 118, ANC 2K.
  2. How long has the patient been receiving Solaris?
    A: Five years, but the patient has periods without treatment due to insurance issues.
  3. Is the patient transfusion independent?
    A: Yes.
  4. What is the current % PNH?
    A: Similar to prior PNH clone size.
  5. Are there any additional cytogenetic abnormalities noted or another potential etiology for the cytopenias?
    A: None on multiple bone marrow biopsies.
  6. Is there any pertinent family history of marrow dysfunction?
    A: Unknown. Both parents died young in an accident, and there is no other family history.

Given that the patient is transfusion-independent, it is reasonable to start the donor search, but with an ANC 2 and plts >50K, it’s also reasonable to hold off for now until counts worsen.

Disclaimer: ASH does not recommend or endorse any specific tests, physicians, products, procedures, or opinions, and disclaims any representation, warranty, or guaranty as to the same. Reliance on any information provided in this article is solely at your own risk.