Trial Outcomes & Findings for Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias (NCT NCT00061568)

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

Number of participants that experience treatment success at one year following stem cell transplant. Treatment success is defined as full donor type hemoglobin on hemoglobin electrophoresis for patients with sickle cell disease and transfusion-independence for patients with beta-thalassemia.

Mean Myeloid Chimerism Level in participants following stem cell transplant.

Number of participants who developed Acute Graft vs Host Disease (GVHD) Grades I, II, III, IV as defined by CIMBTR criteria for Organ Stages of Acute GVHD. Grades are defined as: Grade I: Skin = Maculopapular rash\< 25% of body surface area (BSA); Liver = Total Bilirubin 2-3 mg/dL; Lower GI = stool output/day is 500-999 mL/day. Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day. Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin \>15 mg/dL; Lower GI = Severe abdominal pain with or without ileus. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

Number of participants who developed Limited or Extensive Chronic Graft vs Host Disease (GVHD). Limited disease is characterized by localized skin involvement and/or evidence of hepatic dysfunction. Limited disease is associated with a favorable outcome without systemic therapy, while extensive disease patients have an unfavorable outcome. Extensive chronic GVHD is defined as GVHD occurring after day 100 that did not meet the definition of limited chronic GVHD. Extensive disease presents either with generalized skin involvement, or with localized skin involvement or hepatic dysfunction plus at least one of the following: Liver histology showing chronic progressive hepatitis, bridging necrosis, or cirrhosis Involvement of the eye (Schirmer's test with less than 5 mm wetting) (see "Diagnosis and classification of Sjögren's syndrome") Involvement of minor salivary glands or oral mucosa (as demonstrated on labial or mucosal biopsy specimen) Involvement of any other target organ

Number of participants with regimen failure. Regimen failure is defined as those participants that experienced graft verses host disease or relapse of sickle cell disease or beta-thalassemia.

Number of participants with disease-free survival, as defined by: alive and free of acute complications related to sickle cell disease or beta-thalassemia.

Number of participants overall survival at year 1 and year 2. Overall survival is defined as participants alive at 1 and 2 years following stem cell transplant.

Number of participants that experienced a transplant related mortality, as defined as death from causes other than relapse (such as: GVHD, toxicity, infection, other and unknown causes).