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Home Bone marrow transplantion New treatment reveals bone marrow transplant as a 'therapeutic option' for SCD

New treatment reveals bone marrow transplant as a 'therapeutic option' for SCD

by Margarida Maia, PhD
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A bone marrow transplant approach that does not use high-dose chemotherapy to prevent an immune response against donor stem cells has high event-free and overall survival rates for patients with sickle cell disease (SCD) treated in a phase 2 clinical trial It was shown that

This approach also expands potential donor eligibility and uses a nonmyeloablative pretransplant conditioning regimen. That is, it does not kill all the stem cells in the patient's bone marrow to make space for new stem cells. However, it is powerful enough to prevent rejection of donor stem cells, especially in adults.

The event-free survival rate, which refers to patients alive and free of graft failure, approached 83% two years after transplantation, and the two-year overall survival rate was 94%, according to the researchers. Graft failure occurred in 8 of 32 children and none of the 38 adults in the trial.

“We demonstrate a haploidentical BTM that is not myeloablative. [bone marrow transplant] Contains Thiotepa and PTCy [cyclophosphamide] “It is a readily available curative therapy for most adults, including those with organ damage,” the scientists wrote.

The conditioning therapy is also cheaper and easier to administer than “more expensive myeloablative gene therapy or gene editing” approaches, making it a potential treatment option for low-income countries like Africa. added the research team.

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Trial tested new version of less intense conditioning therapy

the study, “International learning collaborative phase 2 trial of haploidentical bone marrow transplantation in sickle cell disease” was published in the journal by an international group of researchers. blood.

SCD is a genetic disorder that causes red blood cells to become sickle-shaped. This change is caused by the presence of an abnormal form of hemoglobin, a protein in red blood cells that carries oxygen throughout the body, and the symptoms of the disease can be life-threatening.

Allogeneic stem cell transplantation uses donor stem cells to replace a patient's hematopoietic stem cells in the bone marrow. The transplanted cells then begin producing new, healthy red blood cells.

Allogeneic stem cell transplantation offers a treatment for SCD, but its use may be limited by the difficulty of finding compatible donors and the aggressiveness of myeloablative conditioning therapy. These therapies help prevent a patient's body from rejecting transplanted stem cells, but they can be too stimulating for some people and can cause serious complications or death.

“Non-myeloablative or less intense conditioning therapies have been developed specifically for adults with SCD to reduce transplant-related toxicity and mortality,” the researchers wrote. He pointed out that there are major limitations to transplantation. One such regimen The transplant failure rate was 40%.

Scientists led by a team from Vanderbilt University in Nashville Phase 2 study (NCT01850108) To evaluate alternative conditioning therapies and stem cells from haploidentical donors, we evaluate less stringent matching systems that by definition include the patient's family. An alternative regimen combines the chemotherapeutic agent thiotepa with a previously tested reduced-intensity regimen (fludarabine, cyclophosphamide, antithymocyte globulin, and low-dose total body radiation) given in preparation for transplantation in SCD patients. was to be added.

The patient was given cyclophosphamide again after the transplant to prevent graft-versus-host disease, which can occur when stem cells (the graft) attack the patient's body (the host).

Stem cell donors were family members of all 70 study patients.

The study enrolled 70 children and adults diagnosed with SCD, with patients recruited at sites in North America, Europe, and Asia. The median age of this group was 19.1 years, and most (95.6%) received a transplant from a parent or sibling. Graft failure occurred in 8 patients (11.4%), all of whom were younger than 18 years.

“No adults were excluded from the study due to stroke or severe pre-existing heart, lung, or kidney disease,” the scientists noted.

The researchers expected that at least 80% of patients would survive without graft rejection within two years after transplantation. The study's actual event-free survival rate was 82.6% at 2 years, and the overall 2-year survival rate was 94.1%, with “no difference between children and adults,” the researchers wrote.

Most patients had successful engraftment. This means that the transplanted stem cells survived and multiplied within the bone marrow. Of her 59 patients with complete engraftment, 57 (96.6%) discontinued immunosuppressive drugs 1 year after transplantation.

Complications included severe or very severe acute graft-versus-host disease in approximately 10% of patients at 1 year posttransplant, which also did not differ between children and adults (12.5% and 7.9%). At 2 years of follow-up, a similar proportion had moderate or severe chronic graft-versus-host disease. Five patients (7.1%) died from infection.

“To our knowledge, we have completed the first multicenter international phase 2 trial of non-myeloablative haploidentical BTM with thiotepa and PTCy to treat children and adults with SCD,” researchers said. wrote, adding that they believe this approach is “readily available” for everyone. There are SCD patients living in middle- and high-income countries, and “emerging” patients in Africa, where “at least 70% of the world's children with SCD are born.”

This type of bone marrow transplantation and its conditioning regimen “now represents a therapeutic option for adult SCD patients in middle- and high-income BMT centers,” the researchers concluded.

However, in children, the researchers added, “further modifications to conditioning regimens are needed to reduce non-death-related graft failure.”

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Welcome to Daily Transplant News, your trusted source for the latest updates, stories, and information on transplantation and organ donations. We are passionate about sharing the inspiring journeys, groundbreaking research, and invaluable resources surrounding the world of transplantation.

About Us

Welcome to Daily Transplant News, your trusted source for the latest updates, stories, and information on transplantation and organ donations. We are passionate about sharing the inspiring journeys, groundbreaking research, and invaluable resources surrounding the world of transplantation.

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