Evaluating blood or marrow transplantation with post-transplant cyclophosphamide in chronic lymphocytic leukemia

This study aimed to investigate the effect of post-transplant cyclophosphamide on allogeneic blood or marrow transplant in patients with chronic lymphocytic leukemia.  

This study concluded that this treatment approach is safe and effective for these patients.  

Allogeneic blood or marrow transplantation (alloBMT) is a treatment where a patient receives blood or bone marrow from a donor. The blood or marrow helps the patient to create new healthy cells after cancer cells have been destroyed during cancer treatment. A haploidentical transplant is a type of allogeneic transplant. It's also called a half-matched transplant as the donor is a half match for the patient. AlloBMT is the only treatment for chronic lymphocytic leukemia (CLL) with curative potential.  

Cyclophosphamide (Cytoxan) can be given after transplant (post-transplant cyclophosphamide; PTCy) to help decrease the occurrence of graft-versus-host disease (GVHD). GVHD is a complication of transplant where the transplanted cells see the body as foreign and attack it.  

The effect of PTCy on alloBMT outcomes in patients with CLL is still unknown. 

This study involved 64 patients who had CLL. All patients underwent nonmyeloablative (NMA) haploidentical alloBMT. NMA is therapy given before the transplant to kill cancer cells. It is less toxic for cells than regular conditioning and better tolerated. Patients were followed-up for an average of 4.4 years. 

The 4-year overall survival (OS) rate was 52% for all patients. The 4-year progression-free survival (PFS) rate was 37%.  

6 patients suffered from engraftment failure. This is a complication of transplant where the patients do not recover normal bone marrow function. PTCy was associated with lower GVHD occurrence. After 1 year, 27% of patients experienced grade 2 to 4 short-term GVHD. After 2 years, 17% of patients experienced long-lasting GVHD.  

This study concluded that NMA haploidentical alloBMT with PTCy is a safe and effective treatment option for patients with CLL.  

This study had a very small number of participants. Also, the results ware based on medical records. Information might have been missing. Further larger studies are needed for stronger evidence.