Search / Trial NCT00001626

Comparing Therapies for the Treatment of Severe Aplastic Anemia

Launched by NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (NHLBI) · Nov 3, 1999

Trial Information

Current as of October 18, 2024

Completed

Keywords

Severe Aplastic Anemia Immunosuppression

Description

Severe aplastic anemia (SAA) is a disorder with a poor prognosis if untreated. Current accepted therapeutic strategies include bone marrow transplantation (BMT) and immunosuppression, both offering cure or amelioration in the majority of patients. Although BMT is successful using human leukocyte antigen (HLA) matched sibling bone marrow, the 25% probability of finding an HLA identical sibling within a family renders this approach available to only a minority of patients. BMT utilizing HLA-matched, unrelated donors carries a high risk of treatment failure along with considerable toxicity. Wh...

Gender

ALL

Eligibility criteria

  • * INCLUSION CRITERIA:
  • Severe aplastic anemia confirmed at NIH by:
  • 1. Bone marrow cellularity less than thirty percent (excluding lymphocytes).
  • 2. At least two of the following:
  • Absolute neutrophil count less that 500/mm(3);
  • Platelet count less than 20,000/mm(3);
  • Reticulocyte count less than 60,000/mm(3).
  • EXCLUSION CRITERIA:
  • Serum creatinine greater than to 2.5 mg/dl.
  • Cardiac ejection fraction less than 45% by MUGA.
  • Underlying carcinoma (except local cervical, basal cell, squamous cell or melanoma).
  • Current pregnancy or unwilling to take oral contraceptives.
  • Diagnosis of Fanconi anemia or other congenital bone marrow failure syndromes.
  • Evidence of a clonal disorder on cytogenetics.
  • HIV positivity.
  • Inability to understand the investigational nature of the study.
  • Patients who are moribund or have hepatic, renal, cardiac, metabolic or other concurrent diseases of such severity that death within 7-10 days is likely.
  • Previous treatment with ATG, or cyclophosphamide.

About National Heart, Lung, And Blood Institute (Nhlbi)

The National Heart, Lung, and Blood Institute (NHLBI) is a leading component of the National Institutes of Health (NIH), dedicated to advancing research and clinical trials focused on cardiovascular, pulmonary, and hematologic diseases. With a mission to improve public health through innovative research, the NHLBI supports a wide range of studies aimed at understanding, preventing, and treating heart and lung conditions. By collaborating with academic institutions, healthcare providers, and patient communities, the NHLBI strives to translate scientific discoveries into effective clinical practices, ultimately enhancing the quality of life for individuals affected by these critical health issues.

Locations

Bethesda, Maryland, United States

People applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Discussion 0