In a nutshell
This study aimed to investigate the use of stem cell transplant with a chemotherapy treatment regime in patients with acute myeloid leukemia.
This study concluded that this regime is safe and effective in patients that receive stem cell transplants from a haploidentical (half or partially matched) donor.
The use of allogeneic stem cell transplantation (SCT, with a matched donor) in blood cancers that have not responded to previous treatments, such as refractory acute myelogenous leukemia (AML), leads to poor results. is a type of RHM. Sequential treatment strategies have shown good results in AML but have not been validated in the haploidentical transplant setting. Sequential treatment involves the use of more than one treatment. Thiotepa (Thioplex) is a chemotherapy drug that can be used as part of sequential treatment.
It was not known if a thiotepa-based conditioning regime with haploidentical stem cell transplant would be safe and effective in patients with AML.
Methods & findings
This study involved 72 patients. 44 had AML. 7 had ALL. 15 had myelodysplastic syndrome/myeloproliferative neoplasms. 6 had lymphomas. 27 patients received SCT from haploidentical donors (a half matched donor, usually a parent or child). 16 patients received SCT from matched donors (usually a sibling). 29 patients received SCT from unrelated donors. Patients were followed for an average of 21 months.
Patients received sequential treatment that combined chemotherapy and broad antitumor activity. Thiotepa, etoposide (Etopophos) and cyclophosphamide (Cytoxan) were the chemotherapies used. These are administered for 5 days. After 3 days of rest, patients received a reduced intensity conditioning regimen. It involved fludarabine (Fludara), busulfan (Bulsufex) and Thymoglobulin (an immunosuppressant) for 4 days. High-dose post-transplantation cyclophasmide was used for patients who received SCT from haploidentical donors.
The 2-year overall survival (OS, time from treatment until death from any cause) was 54.7% for haploidentical recipients. The 2-year event-free survival (EFS, time from treatment until an event such as disease relapse) was 49.3% for haploidentical recipients.
The OS was 49.2% for matched related recipients. The EFS was 43.8% for matched related recipients.
The OS was 37.9% for unrelated recipients. The EFS was 28% for unrelated recipients.
The incidence of graft versus host disease (GVHD, when the transplanted cells attack healthy organs) was 41.4% in patients who received unrelated SCT compared to 11.1% for patients who received haploidentical SCT.
The incidence of GVHD-free, relapse-free survival was 44.4% for patients who received unrelated SCT compared to 10.3% for patients who received haploidentical SCT.
The bottom line
This study concluded that a thiotepa-based sequential approach with allogenic SCT in patients who receive SCT from a haploidentical donor was a safe and effective treatment regime for AML patients.
Consult your physician about sequential conditioning regimes that may be available to you.
Published By :
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Jan 11, 2018