Table 8.

Bone marrow/stem cell transplantation for patients with PNH

Indications for consideration of transplantation 

  • Bone marrow failure

    • Decision on transplantation is based on underlying marrow abnormality (eg, aplastic anemia)*

  • Major complication of PNH†

    • Recurrent, life-threatening thromboembolic disease

    • Refractory, transfusion-dependent hemolytic anemia‡

 
PNH-specific transplant-related issues 

  • The conditioning regimen of cyclophosphamide/ATG is recommended for patients with PNH/aplastic anemia. For patients with classic PNH, a more myeloablative regimen is indicated.§

  • Additional investigation is required to define the role of nonmyeloablative regimens.

  • For syngeneic twin transplants, a myeloablative conditioning regimen is recommended to prevent recurrence of PNH.

  • There are no PNH-specific adverse events associated with transplantation; severe, acute graft-versus-host disease (GVHD) occurs in more than a third of the patients and the incidence of chronic GVHD is roughly 35%.

  • Overall survival for unselected PNH patients who undergo transplantation using an HLA-matched sibling donor is in the range of 50% to 60%.

 
Indications for consideration of transplantation 

  • Bone marrow failure

    • Decision on transplantation is based on underlying marrow abnormality (eg, aplastic anemia)*

  • Major complication of PNH†

    • Recurrent, life-threatening thromboembolic disease

    • Refractory, transfusion-dependent hemolytic anemia‡

 
PNH-specific transplant-related issues 

  • The conditioning regimen of cyclophosphamide/ATG is recommended for patients with PNH/aplastic anemia. For patients with classic PNH, a more myeloablative regimen is indicated.§

  • Additional investigation is required to define the role of nonmyeloablative regimens.

  • For syngeneic twin transplants, a myeloablative conditioning regimen is recommended to prevent recurrence of PNH.

  • There are no PNH-specific adverse events associated with transplantation; severe, acute graft-versus-host disease (GVHD) occurs in more than a third of the patients and the incidence of chronic GVHD is roughly 35%.

  • Overall survival for unselected PNH patients who undergo transplantation using an HLA-matched sibling donor is in the range of 50% to 60%.

 
*

If the dominant clinical abnormalities are a consequence of bone marrow failure (eg, hypoproliferative anemia, neutropenia with recurrent infections, thrombocytopenia with major bleeding complications) the decision to recommend transplantation should be based on guidelines for management of the specific marrow failure syndrome.

†

Factors including age, comorbid conditions, availability of an HLA-matched sibling donor, and time from initial diagnosis should be considered before recommending transplantation for major complications of PNH.

‡

The availability of new treatment options (eg, eculizumab) may influence the decision to recommend transplantation.

§

Radiation- or busulfan-based.

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