Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders

Masonic Cancer Center, University of Minnesota1 个研究点分布在 1 个国家目标入组 10 人开始时间: 2000年6月最近更新: 2017年12月28日

适应症

干预措施

试验速览

阶段: 2 期
状态: 已完成
发起方: Masonic Cancer Center, University of Minnesota
入组人数: 10
试验地点: 1
主要终点: Number of Patients Alive (Survival) at 2 Years

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.

详细描述

Prior to transplantation, subjects will receive the drugs busulfan (orally or through the catheter), as well as fludarabine and anti-thymocyte globulin (ATG) via the catheter. Busulfan, fludarabine and ATG will be given with Total Lymphoid Irradiation (TLI) to help the new donor bone marrow take and grow after transplantation.

Those patients receiving donor marrow will have the T cells (a type of white blood cell in the donor marrow) removed to lower the risk that the new marrow will react to their body, a condition called Graft-Versus-Host-Disease (GVHD). After bone marrow transplantation, subjects will receive drugs to help prevent GVHD, including cyclosporin and mycophenolate mofetil (MMF).

Blood samples are taken at day 28, day 60, day 100, 1 year and as required by medical status yearly for five years after transplant to evaluate how well the new marrow is growing. A bone marrow biopsy is required at day 21, at day 100 and 1 year.

研究设计

研究类型: Interventional
分配方式: Non Randomized
干预模型: Parallel
主要目的: Treatment
盲法: None

入排标准

年龄范围: — 至 35 Years（Child, Adult）
性别: All
接受健康志愿者: 否

入选标准

•Patients eligible for transplantation under this protocol will be <35 years of age, and will be diagnosed with:
•a bone marrow failure syndrome unresponsive to available therapy, including but not limited to Diamond-Blackfan anemia, Shwachman Diamond syndrome or Kostmann's neutropenia but exclusive of aplastic anemia.
•Diamond Blackfan Anemia:
•Patients must show evidence of steroid resistance requiring equivalent of >6 transfusions yearly despite steroid therapy.
•Evidence of developing aplasia or myelodysplasia will also be criteria for transplantation.
•Kostmann's Neutropenia, Shwachman-Diamond syndrome:
•Patients must have been previously diagnosed as having a clinical picture characteristic of Shwachman-Diamond syndrome (exocrine pancreatic insufficiency, growth retardation, metaphyseal dysostosis, neutropenia), or must have a bone marrow aspirate consistent with Kostmann's neutropenia, with no evidence of acute leukemia.
•Patients must have failed therapy with granulocyte-colony stimulating factor (G-CSF), as determined by an inability to maintain an absolute neutrophil count (ANC) >750 cells/ml(3), or manifesting recurrent infections despite G-CSF administration resulting in life threatening infections or repeated hospitalizations (<4 /year).

排除标准

•Patients >35 years of age
•Karnofsky score <70%
•Hepatic dysfunction as determined by bilirubin >3.0, ALT >150, or active hepatitis
•Pulmonary function tests with forced volume vital capacity (FVC) and forced expiratory volume (FEV) <70%; O2 saturation <94%
•Renal dysfunction with glomerular filtration rate (GFR) <30% of predicted.
•Cardiac compromise, with left ejection fraction <45%.
•Severe, stable neurologic impairment.
•Human immunodeficiency virus (HIV) positivity.
•Pregnant or lactating females