Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia

Inclusion criteria:

• All patients must:

  • Have related donors who are identical at 6 human leukocyte antigens (HLA) loci (A, B and DR) by molecular typing
  • Have a performance status from 0-2
  • Give written informed consent

• Patients with sickle cell disease should have 1 or more of the following:

  • Acute chest syndrome requiring recurrent hospitalization or exchange transfusion
  • Nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours
  • Recurrent vaso-occlusive pain (2 episodes or more per year) or recurrent priapism
  • Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate 30-50 percent of normal predicted value)
  • Bilateral proliferative retinopathy and major visual impairment in at least 1 eye
  • Osteonecrosis of multiple joints

• Patients with thalassemia should have 1 or more of the following:

  • Transfusion dependence, defined as a transfusion requirement of greater than or equal to 6 units of packed red blood cells over the past 12 months
  • Iron overload, defined as serum ferritin greater than 500 mcg/L in the absence of infection or biopsy-proven iron overload
  • Presence of 2 or more alloantibodies against red cell antigens

Exclusion criteria:

• Pregnancy
• Acute hepatitis (transaminases greater than 3 times the normal value)
• Cardiac ejection fraction less than 30 percent
• Severe renal impairment (glomerular filtration rate less than 30 percent of predicted normal value)
• Severe residual functional neurologic impairment (other than hemiplegia alone)
• Seropositivity for the human immunodeficiency virus (HIV)