Stem Cell Transplant Treatment Protocols

Treatment study protocols describe exactly how the physicians and staff at Lurie Children’s will provide treatment to the patients, from pre-treatment to transplant and follow-up. These protocols provide information on the exact regimen of drugs to be given as conditioning therapy prior to transplantation and on the recommendations for supportive care prior to and after transplantation.  Treatment clinical trials currently available include:

Full Intensity: This investigator-driven protocol outlines the full intensity treatment given for patients with leukemia, MDS or lymphoma. It is standard treatment care for patients who are not otherwise eligible for experimental therapies, as well as those who prefer the current standard of care. Learn more about this study.

Reduced Intensity Transplant: This investigator-driven protocol is a reduced intensity conditioning protocol for related and unrelated hematopoietic stem cell transplantation. This approach can minimize the acute and chronic toxicities of transplantation. Learn more about this study.

SCIDS: This investigator-driven study provides a reduced intensity transplant to patients with Severe Combined Immunodeficiency. A reduced intensity transplant is given to hopefully lead to the correction of the underlying immunological defects, while causing less complications than a full intensity transplant. Learn more about this study.

Reduced Toxicity: This investigator-driven study looks to provide reduced intensity conditioning prior to hematopoietic stem cell transplant to reduce short- and long-term toxicities that can occur after stem cell transplantation. Its goal is to reduce morbidity and mortality by reducing the intensity of pretransplant chemotherapy. Learn more about this study.

Sickle Cell: This multisite study is a full intensity protocol. Unrelated but closely matched donors are used as the stem cell source for patients with severe sickle cell disease. Learn more about this study.

Reduced Intensity Transplant with ECP: This investigator-driven protocol studies the use of extracorporeal photopheresis (ECP) in combination with a reduced intensity conditioning protocol. This approach may minimize some acute and chronic toxicities of transplantation. Learn more about this study.

StemEx: This industry-sponsored protocol provides an alternative to a typical stem cell transplant for patients with certain types of leukemia and lymphoma who are unable to find a matched donor. This full-intensity transplant involves transplantation of a graft of expanded stem/progenitor cells from a single cord blood unit, as well as unmanipulated cells from the same cord blood unit. Learn more about this study.

Autoimmune Disorders: This multisite study looks at the use of autologous stem cell rescue for patients with autoimmune disease. It provides transplant for patients with severe disease who have failed several different lines of alternate treatment. Learn more about this study.

Recurrent/Refractory Brain Tumors: This consortium study involves recurrent/refractory brain tumor patients from a number of clinical sites. This protocol involves treatment both before and after an autologous stem cell transplant. Learn more about this study.

Double Transplant for Lymphoma: This study is open at several sites, headed by Columbia University. It involves an autologous transplant, followed by a reduced intensity allogeneic transplant.  The protocol is specifically for patients with relapsed or refractory Hodgkin's disease or non-Hodgkin's lymphoma. Learn more about this study.

Solid Tumor: Tandem autologous stem cell rescues are given to children with recurrent solid tumors. This investigator-driven protocol incorporates the use of double sequential high-dose therapy regimens to treat patients who achieve a second complete remission or a partial response after a solid tumor. Patients determined to have chemo sensitive disease are eligible. Learn more about this study.

Palifermin: This industry-sponsored study looks at the safety and appropriate dosing of Palifermin for pediatric subjects with hematologic malignancies undergoing allogeneic transplantation. Future studies focusing on efficacy of Palifermin in reducing mucositis will be based on the dose level of Palifermin established in this study. Learn more about this study.

All protocols are approved by the Lurie Children's Institutional Review Board.