Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

  • STATUS
    Recruiting
  • participants needed
    40
  • sponsor
    University of Florida
Updated on 12 August 2022
malignant disease
hysterectomy
stem cell transplantation
graft versus host disease
fludarabine
anemia
immunodeficiency
busulfan
anti-thymocyte globulin
ejection fraction
deficiency
lfts
cell transplantation
t-cell depletion
bone marrow procedure
gilbert's syndrome
transplant conditioning
thiotepa
shortening fraction
antithymocyte globulin
cytopenia
thrombocytopenia
hemolysis
replacement therapy
follicle stimulating hormone
malnutrition
metabolic disorders
hurler's syndrome
thrombocytopenia, congenital
pulse oximetry
bilateral tubal ligation
follicle-stimulating hormone
conditioning regimen
lymphoproliferative disorder
diamond-blackfan anemia
neutropenia
hemoglobinopathy
undernutrition
sickle cell anemia
hla typing
x-linked lymphoproliferative disease
lymphohistiocytosis
wiskott-aldrich syndrome
lymphoproliferative disease
hemophagocytic lymphohistiocytosis
thalassemia
ratg
familial hemophagocytic lymphohistiocytosis
anti-thymocyte globulin (rabbit)

Summary

In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.

Description

Hematopoietic stem cell transplantation is the only curative choice for a number of inherited bone marrow failure syndromes, hemoglobinopathies, metabolic disorders and primary immune deficiencies. While survival of these patients is typically better than survival of patients with malignancies, toxicities of conditioning regimens and failure of engraftment remain challenges. Most children with non-malignant disorders present with normocellular or even hypercellular bone marrow, posing a barrier to engraftment and requiring intensive conditioning. Commonly used backbone of busulfan and fludarabine, although well tolerated, results in variable engraftment, in particular with mismatched unrelated donors and cord blood recipients. In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) in order to determine the minimum effective dose required for reliable engraftment. Subjects are stratified in groups A and B based the risk of graft failure.

Details
Condition Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Treatment Thiotepa--single daily dose, Thiotepa--escalated dose
Clinical Study IdentifierNCT03513328
SponsorUniversity of Florida
Last Modified on12 August 2022

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