Overview

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

Status:
Completed
Trial end date:
1969-12-31
Target enrollment:
0
Participant gender:
All
Summary
OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders. II. Determine the quality of life, absence of adverse effects (e.g., graft versus host disease and B cell lymphoproliferative disease), and completeness of recovery of their underlying condition in these patients with this treatment regimen.
Phase:
N/A
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Fairview University Medical Center
Treatments:
Antilymphocyte Serum
Busulfan
Cyclophosphamide
Criteria
PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Severe hemoglobinopathy including, but not limited to the following:

Homozygous beta thalassemia Other beta chain mutation as demonstrated by hemoglobin
electrophoresis Sickle cell anemia (age 16 to 50 years) Evidence of one or more the
following prior complications: Stage I-II sickle cell lung disease Sickle cell nephropathy
(moderate to severe proteinuria or glomerular filtration rate of 30-50% predicted normal
for age) Acute chest syndrome requiring blood transfusions More than 3 debilitating pain
episodes per year for 3 years lasting more than 4 hours each Any combination of episodes of
acute chest syndrome and painful episodes adding up to 3 episodes per year for 3
consecutive years Requirement for chronic transfusions with alloimmunization (more than 2
antibodies) Sickle cell anemia (age under 16 years) Evidence of one or more the following
prior complications: Prior stroke or hemorrhage Any neurologic event lasting more than 24
hours Abnormal cerebral MRI and cerebral arteriogram MRI angiographic impaired
neuropsychologic testing Stage I-II sickle cell lung disease Sickle cell nephropathy
(moderate to severe proteinuria or glomerular filtration rate of 30-50% predicted normal
for age) Significant visual impairment in at least one eye with bilateral proliferative
retinopathy Acute chest syndrome with history of recurrent hospitalizations or exchange
transfusions Osteonecrosis of multiple joints with destructive changes More than 3
debilitating pain episodes per year or priapism Requirement for chronic transfusions with
alloimmunization

OR

Bone marrow failure syndrome unresponsive to therapy, including but not limited to the
following: Congenital pure red cell aplasia (Diamond Blackfan anemia) Confirmed by bone
marrow aspirate More than 6 transfusions per year despite steroid therapy Kostmann's
neutropenia Confirmed by bone marrow aspirate Unable to maintain absolute neutrophil count
greater than 750/mm3 or recurrent life threatening infections or more than 4
hospitalizations per year despite therapy with filgrastim (G-CSF) No myelodysplasia or
aplastic anemia

Must have a related donor with at least a 5 out of 6 HLA antigen match

--Patient Characteristics--

Age: Sickle cell anemia patients 0 to 50; All other patients under 18

Performance status: Karnofsky 70-100%

Hepatic: Bilirubin no greater than 3.0 mg/dL with a direct fraction no greater than 2.0
mg/dL ALT no greater than 150 U/L No moderate or severe portal fibrosis No active hepatitis

Renal: Glomerular filtration rate at least 30% predicted (except with sickle cell anemia)
No renal dysfunction

Cardiovascular: Left ejection fraction at least 50% No cardiac compromise

Other: No severe, stable neurologic impairment HIV negative