Researchers at Vanderbilt University Medical Center (VUMC) have highlighted promising results from a phase II clinical trial for a new treatment for sickle cell disease (SCD). Published in Blood, the study aimed to achieve an 80% two-year event-free survival rate following a specific bone marrow transplant (BMT) method. The international trial, led by Dr DeBaun and Dr Adetola Kassim, involved 32 children and 38 adults from five countries.
The treatment protocol, which included thiotepa and post-transplant cyclophosphamide (PTCy), showed that 11.4% of participants experienced graft failure, all of whom were children. Despite this, those with successful engraftment had excellent outcomes, with a median donor chimerism of 100% at 180 and 365 days post-transplant. One year after the transplant, 96.6% of these patients were off immunosuppression. Both acute and chronic severe graft-versus-host disease (GVHD) rates were low at 10%. However, five participants (7.1%) died from infections.
The overall two-year survival rate was about 95% for both children and adults. All children who experienced graft failure recovered without needing additional treatment. Further research is ongoing to improve engraftment in children, while the protocol for adults remains effective.
Previously, curative options for adults with SCD were limited due to the need for fully matched donors and the harshness of myeloablative treatment. The new haploidentical transplant protocol, which requires only a half-match from a relative, preserves fertility and is accessible to 90% of potential participants.
This treatment is more accessible and affordable than FDA-approved gene therapies, which cost between $2 million and $3 million. Most transplant centers can immediately implement this protocol, making it a viable option worldwide.
“We are offering another curative option for SCD that is both safe and more affordable compared to recently FDA-approved gene therapy and gene editing trials,” said Dr. DeBaun. “It is crucial for physicians and families to be aware of our findings to make informed decisions about the best personalized curative therapy options.“
Disclosures: This article was created by the touchHAEMATOLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
