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

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

Summary

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.

Description

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

Conditions

Non Malignant Diseases

Intervention

Hematopoetic Stem Cell Transplant using Reduced Intensity Conditioning Regimen

Location

The Children's Hospital of Philadelphia
Philadelphia
Pennsylvania
United States
19104

Status

Recruiting

Source

Children's Hospital of Philadelphia

Results (where available)

View Results

Links

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

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Study of Low-Intensity Conditioning for Allogeneic Stem Cell Transplant

The purpose of this study is to determine whether a reduced intensity conditioning regimen for stem cell transplant with donor cells will allow the donor cells to be effective without caus...

Immunotherapy Following Reduced Intensity Conditioning and Allogeneic Stem Cell Transplant for Poor Risk CD30+ Hodgkin Lymphoma Patients

Patients with relapsed or refractory Hodgkin Lymphoma who are CD30+ will receive a standard of care reduced intensity regimen and an allogeneic stem cell transplant (from another person, r...

Reduced-Intensity Conditioning (RIC) and Myeloablative Conditioning (MAC) for HSCT in AML/MDS

The purpose of this study is to compare safety and efficacy of reduced-intensity conditioning and myeloablative conditioning regimens prior to HSCT in high-risk AML/MDS pediatric and young...

MiniMUD Study - Unrelated Reduced Intensity Conditioning With Treosulfan® for Allogeneic Stem Cell Transplantation in Patients With Hematological Malignancies

In this study, treosulfan is evaluated for conditioning in allogenic stem cell transplantation. The procedure and the follow-up are the same as in standard allogenic transplant. The...

A Trial of Extracorporeal Photopheresis, Pentostatin, and Total Body Irradiation in Patients Undergoing Reduced Intensity Allogeneic Stem Cell Transplantation for the Treatment of Malignancies

This is a study to explore the use of a reduced intensity transplant conditioning regimen. A conditioning regimen is the treatment that is given to prepare a body for the new bone marrow t...

PubMed Articles [25720 Associated PubMed Articles listed on BioPortfolio]

Thiotepa 10 mg/kg Treatment Regimen Is Superior to Thiotepa 5 mg/kg in TBF Conditioning in Patients Undergoing Allogeneic Stem-Cell Transplantation.

The optimal intensity of myeloablation with a reduced-toxicity conditioning regimen to decrease relapse rate after allogeneic stem-cell transplantation without increasing transplant-related mortality ...

Infusion of cytotoxic T lymphocytes for the treatment of viral infections in hematopoetic stem cell transplant patients.

Allogeneic hematopoietic stem cell transplantation has proven curative for a range of malignant and nonmalignant disorders. However, the clinical success of this therapy is marred by the morbidity ass...

Outcomes of reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation for 10 patients with myelofibrosis.

To evaluate the efficacy of reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation (RIC-allo-HSCT) for patients with myelofibrosis (MF).The clinical data of 10 patients with...

Autologous stem cell transplant for AL amyloidosis patients aged 70 to 75.

Autologous stem cell transplantation has been used in treatment for AL amyloidosis for over two decades and generally reserved for patients younger than 70 years of age. Herein we report on outcomes o...

Higher Donor Apheresis Blood Volumes are Associated with a Reduction in Relapse Risk and Improved Survival in Reduced Intensity Allogeneic Transplants with Unrelated Donors.

Allogeneic hematopoietic stem cell transplantation (HSCT) with reduced intensity conditioning (RIC) offers a curative option for patients with hematologic malignancies who are unable to undergo myeloa...

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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