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OBJECTIVES: I. Determine the safety of cyclosporine and mycophenolate mofetil as a non-ablative conditioning and post-transplantation immunosuppression regimen in patients with primary T-cell immunodeficiency disorders who undergo HLA-matched related or unrelated bone marrow transplantation to induce mixed hematopoietic chimerism (establishment of 1-95% donor CD3+ cells).
II. Determine the kinetics of immune reconstitution of lymphoid cell subsets, T-cell function, and B-cell function after allogeneic bone marrow transplantation in this patient population.
PROTOCOL OUTLINE: Patients are stratified according to type of primary T-cell immunodeficiency disorder (severe combined immunodeficiency syndrome (SCID) vs non-SCID) and donor status (related vs unrelated).
All patients receive cyclosporine orally or IV on days -1 through 50 and oral mycophenolate mofetil on days 0 through 27 in the absence of unacceptable toxicity. Unrelated donor recipients and non-SCID patients also undergo total body irradiation on day 0. All patients then undergo allogeneic bone marrow transplantation on day 0. Cyclosporine taper regimen begins on day 50 and continues through day 180 unless evidence of graft-versus-host disease.
Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Purine-Pyrimidine Metabolism, Inborn Errors
Cyclosporine, Mycophenolate mofetil
Fred Hutchinson Cancer Research Center
Office of Rare Diseases (ORD)
Published on BioPortfolio: 2014-07-23T21:56:13-0400
Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders
OBJECTIVES: I. Determine the safety of total body irradiation and fludarabine followed by allogeneic peripheral blood stem cell or bone marrow transplantation in combination with cyclospor...
Psoriasis is an immuno-mediated skin disorder, which affects about 2-3% of the population worldwide. For moderate-severe forms of psoriasis systemic immunosuppression is the treatment of c...
This is a retrospective study aimed at establishing a database of the current health of adult patients with IEM in the French-speaking part of Switzerland. .
Study Comparing Efficacy and Safety of Mycophenolate Mofetil (Cellcept) With Delayed Introduction of Sirolimus and Discontinuation of Cyclosporine, With Those of Mycophenolate Mofetil and Long Term Continuation of Cyclosporine in Renal Transplant Recipien
This multicentre, prospective, randomized, open-label study will compare the safety and efficacy of mycophenolate mofetil with delayed introduction of sirolimus and discontinuation of cycl...
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabo...
Many inborn errors of metabolism may present with epilepsy or seizures, however, current scope of these diseases is unknown. Due to available precision medicine approaches in many inborn errors of met...
We propose a nosology for inborn errors of metabolism that builds on their recent redefinition.
Endocrinologists may encounter abnormal results in routine laboratory tests while caring for patients with inborn errors of metabolism. This article provides a framework for understanding these abnorm...
Inborn errors of metabolism (IEMs) are disorders with various manifestations that occur mainly in the pediatric population. In countries where consanguineous marriage is common, the association betwee...
The use of medical foods (MFs) specifically for non-inborn errors of metabolism (non-IEM) is rising. Concomitantly, evidence for the safety and efficacy of these non-IEM MFs is lacking. We examined th...
Dysfunctions in the metabolism of PURINES or PYRIMIDINES resulting from inborn genetic mutations that are inherited or acquired in utero.
Errors in the metabolism of LIPIDS resulting from inborn genetic MUTATIONS that are heritable.
Errors in metabolic processes resulting from inborn genetic mutations that are inherited or acquired in utero.
Errors in metabolic processing of STEROIDS resulting from inborn genetic mutations that are inherited or acquired in utero.
Dysfunctions in the metabolism of metals resulting from inborn genetic mutations that are inherited or acquired in utero.
Organ transplantation is the moving of an organ from one body to another or from a donor site to another location on the patient's own body, for the purpose of replacing the recipient's damaged or absent organ. The emerging field of regenerative ...
Osteoporosis is a disease in which the bones become extremely porous, are subject to fracture, and heal slowly, occurring especially in women following menopause and often leading to curvature of the spine from vertebral collapse. Follow and track&n...