Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

2014-08-27 03:16:34 | BioPortfolio


The purpose of this study is to see if we can engraft (grow) donor cells and cure the underlying disease by using Reduced Intensity Conditioning (RIC) before receiving the donor blood or marrow cells. RIC involves giving medicines that suppress the immune system before giving the donor stem cells. It does not completely eliminate blood making cells in bone marrow. This is in contrast to standard myeloablative conditioning that would completely "wipe out" bone marrow with high doses of chemotherapy and radiation. The possible benefits of a reduced intensity conditioning regimen are fewer short-term toxicities of lungs, liver and heart, and less long-term side effects. The hospital stay is usually shorter than after conditioning that completely destroys the bone marrow, and blood counts may recover more quickly. In general, the benefits of undertaking this transplant include possible cure of primary disease.


Allogeneic hematopoietic stem cell transplant (HSCT) is curative for many non-malignant diseases, including immunodeficiencies and hemoglobinopathies. Standard myeloablative therapy to establish complete donor chimerism usually includes busulfan, cyclophosphamide +/- thymoglobulin. This approach may result in full donor engraftment with complete immune reconstitution. However, this approach has been associated with a poor outcome in patients with significant organ dysfunction, and is associated with significant organ morbidities including veno-occlusive disease. Late complications include gonadal failure and secondary malignancies.

Recently, reduced intensity conditioning (RIC) regimens have been used for both adult patients with leukemias and pediatric patients with non-malignant diseases. These regimens are better tolerated, resulting in less transplant related morbidity and mortality. Stable mixed chimerism, while insufficient for eradication of leukemias, may be sufficient to cure patients with non-malignant diseases.

There are two conditioning regimens in this protocol for children >6 months. They differ by the timing and dosing of Alemtuzumab (Campath). The "distal" Campath regimen proposed is identical to the regimen proposed by the Clinical Trials Network for patients with sickle cell disease with unrelated donors and has been used successfully in patients with IPEX. The "proximal" Campath regimen protocol is used extensively in the UK for immune deficiencies, and is currently in use internationally for HLH and LCH. Autologous reconstitution (graft loss) may be increased with RIC, but life-threatening aplasia from graft failure is rare.

The conditioning regimen for children with immunodeficiencies <6 months omits melphalan, and substitutes two days of busulfan. This regimen is successfully used in the UK, and has been successful in a 3 month old infant at CHOP who engrafted with a haploidentical donor.

Study Design

Control: Uncontrolled, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Non Malignant Diseases


Hematopoetic Stem Cell Transplant using Reduced Intensity Conditioning Regimen


The Children's Hospital of Philadelphia
United States




Children's Hospital of Philadelphia

Results (where available)

View Results


Published on BioPortfolio: 2014-08-27T03:16:34-0400

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Medical and Biotech [MESH] Definitions

Preparative treatment of transplant recipient with various conditioning regimens including radiation, immune sera, chemotherapy, and/or immunosuppressive agents, prior to transplantation. Transplantation conditioning is very common before bone marrow transplantation.

Bone-marrow-derived, non-hematopoietic cells that support HEMATOPOETIC STEM CELLS. They have also been isolated from other organs and tissues such as UMBILICAL CORD BLOOD, umbilical vein subendothelium, and WHARTON JELLY. These cells are considered to be a source of multipotent stem cells because they include subpopulations of mesenchymal stem cells.

Methods of implanting a CELL NUCLEUS from a donor cell into an enucleated acceptor cell. Often the nucleus of a somatic cell is transferred into a recipient OVUM or stem cell (STEM CELLS) with the nucleus removed. This technology may provide means to generate autologous diploid pluripotent cell for therapeutic cloning, and a model for studying NUCLEAR REPROGRAMMING in embryonic stem cells. Nuclear transfer was first accomplished with frog eggs (RANA PIPIENS) and reported in 1952.

The release of stem cells from the bone marrow into the peripheral blood circulation for the purpose of leukapheresis, prior to stem cell transplantation. Hematopoietic growth factors or chemotherapeutic agents often are used to stimulate the mobilization.

The malignant stem cells of TERATOCARCINOMAS, which resemble pluripotent stem cells of the BLASTOCYST INNER CELL MASS. The EC cells can be grown in vitro, and experimentally induced to differentiate. They are used as a model system for studying early embryonic cell differentiation.

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