Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
研究概览
简要总结
OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure syndromes.
II. Assess the efficacy of a reduced maintenance dose in patients who respond to daily G-CSF.
III. Assess the toxic effects of G-CSF in these patients. IV. Measure bone marrow progenitor colonies before and after G-CSF. V. Measure CD34-positive cells in marrow and blood before and after G-CSF using flow cytometry and immunohistochemistry.
详细描述
PROTOCOL OUTLINE: Patients receive granulocyte colony-stimulating factor (G-CSF) subcutaneously every day for 8 weeks; nonresponders receive an increased dose for an additional 8 weeks. Patients who respond at week 8 or 16 are then tapered to a lower maintenance dose of G-CSF administered every other day through week 40. The dose is adjusted to maintain an absolute neutrophil count above 1500.
Patients are removed from study for failure to achieve a complete response by week 16, unacceptable nonhematologic toxicity, the identification of a clonal karyotype in marrow, or the onset of leukemia.
研究设计
- 研究类型
- Interventional
- 主要目的
- Treatment
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •PROTOCOL ENTRY CRITERIA:
- •-Disease Characteristics--
- •Inherited bone marrow failure syndrome, including:
- •Fanconi's anemia
- •Dyskeratosis congenita
- •Shwachman syndrome
- •Amegakaryocytic thrombocytopenia
- •Decreased megakaryocytes in infancy
- •No thrombocytopenia with absent radius syndrome (TAR)
- •No trisomy 13 or 18
- •No clonal bone marrow karyotype
- •-Prior/Concurrent Therapy--
- •At least 4 weeks since growth factors
- •Concurrent therapy allowed if not altered for 30 days prior to entry through week 8
- •No concurrent investigational drugs
- •-Patient Characteristics--
- •Hematopoietic: ANC <1000
- •No leukemia
- •Other: No medical or psychiatric contraindication to protocol participation
- •No pregnant or nursing women
排除标准
- 未提供