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

• Status: Terminated
• Trial end date: 2003-11-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
• Treatments: Busulfan; Fludarabine; Fludarabine phosphate; Prednisone; Tacrolimus

Criteria

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)