Haploidentical Bone Marrow Transplant – Pediatric Oncology at Johns Hopkins University Kimmel Cancer Center

The lack of suitable bone marrow donors to meet patients’ needs inspired Johns Hopkins scientists to pioneer haploidentical bone marrow transplantation (BMT). This breakthrough has made bone marrow transplantation possible for nearly all patients in need of treatment.

With haploidentical BMT, parents, siblings, and even uncles and aunts, nieces and nephews, half-siblings, and grandparents can safely serve as donors. Our experts have performed more than 500 Haprioidentical transplants for leukemia and lymphoma in adults and children, with safety and toxicity comparable to traditional transplants. This safety allows this type of his BMT, available only at Johns Hopkins University, to be used to treat many types of cancer and non-cancerous childhood diseases.

• Pediatric hematological malignancies
• In pediatric solid tumors, it induces a graft-versus-tumor effect and reduces recurrence in high-risk cancers.
• sickle cell anemia
• immune disease
• Used in conjunction with solid organ transplantation to prevent organ rejection and eliminate the risks and costs associated with lifelong treatment with immunosuppressants.

Pediatric bone marrow transplant expert Heather Simmons is leading a clinical study of haploidentical bone marrow transplants for patients with refractory and/or relapsed high-risk leukemia and lymphoma. This study is available only at Johns Hopkins University. Symon’s new trial uses semi-matched donors, making it an option for nearly all patients for whom BMT is indicated. The safety and toxicity of haploidentical bone marrow transplants performed to date are comparable to matched transplants. As a result, this strategy is now used in the initial treatment of childhood leukemias and lymphomas and has been extended to children with solid tumors such as sarcomas and neuroblastomas to promote a graft-versus-tumor effect and eliminate cancer. Preventing recurrence.

Haploidentity BMT Clinical Trial:

Phase II trial of partially HLA-mismatched (HLA-haploidentical) bone marrow transplantation for high-risk solid tumors.

the purpose:

Allogeneic hematopoietic stem cell transplantation (HSCT) may be associated with clinically important “graft-versus-tumor” (GVT) effects, even for diseases that do not respond to chemotherapy or radiotherapy. Graft-versus-tumor (GVT) effects after allogeneic HCT for neuroblastoma, Ewing’s sarcoma, osteosarcoma, rhabdomyosarcoma, melanoma, and hepatoblastoma have been reported. Our goal is to maximize T cell- and NK cell-mediated graft-versus-tumor effects in individuals with poor prognosis. Oncology patients using a haploidentical donor, T cell-rich bone marrow, and a unique post-transplant immunosuppressive regimen including post-transplant Cy and mTOR inhibitors. Because nearly all patients have a semi-compatible donor (parent or sibling), this treatment is likely to be widely applicable. We look forward to combining this platform with additional post-transplant treatments such as cryoablation, donor lymphocyte infusion (DLI), stem cell-directed therapies, immune checkpoint inhibitors, and metabolic inhibitors to improve the safety of this treatment. We want to demonstrate feasibility and feasibility.

Pediatric Blood and Marrow Transplant Consortium (PBMTC) multicenter phase II pilot trial investigates cyclophosphamide-induced myeloablation after transplantation of partially HLA-mismatched T-cell-enriched bone marrow in pediatric patients with hematologic malignancies. We are considering physical conditioning and transplantation.

the purpose:

We propose a multicenter phase II study in children with high-risk leukemia in first CR, acute leukemia in second CR, MDS, and JMML. The myeloablative conditioning therapy prescribed is TBI-based for lymphocytic leukemia, busulfan-based for myeloid leukemia, or busulfan-based for lymphocytic leukemia for which TBI-based therapy was used for the first transplant. It becomes. Our goal is to establish an easily exportable and inexpensive platform for haplotransplantation with a safety profile comparable to matched related and unrelated BMTs. The primary objective is to estimate 6-month non-relapse mortality assuming an NRM of less than 18%.

Bone marrow transplantation and high-dose posttransplant cyclophosphamide for chimerism induction and renal allograft tolerance

the purpose:

Kidney transplantation is a good treatment for patients with end-stage renal disease. However, there is still much to learn about how to properly care for a transplanted kidney and keep it functioning over time. Unless a person who receives a kidney from someone else takes drugs that weaken their immune system, the kidney will be rejected. You must continue taking these medications for the rest of your life. The side effects of these drugs can cause many problems, such as infections and cancer, and often shorten lifespan. Even long-term use of immunosuppressants cannot completely prevent the development of rejection reactions. Episodes of rejection can lead to long-term weakness and loss of the transplanted kidney due to cumulative chronic rejection effects. There is only about a 50% chance that a transplanted kidney will continue to function after 10 years. Receiving dialysis reduces both the quality and duration of life, and subsequent transplantation increases the risk of transplantation due to sensitization. Life expectancy after a second or third kidney transplant is even shorter. For all these reasons, achieving tolerance in the transplanted kidney without chronic rejection and without the need for permanent immunosuppressive treatment is a highly desirable goal. If this becomes a reality, it will become possible to have one kidney for life.

Qualifications:

Patients 18 to 65 years of age receiving their first kidney allograft from an HLA haploidentical living related donor.