Catholic University Catholic Hematology Hospital

Allogeneic hematopoietic stem cell transplantation is the sole curative treatment for patients with myelofibrosis. However, this procedure can be challenging to perform on elderly patients, those in poor general health, or those with other medical conditions. In addition, significant treatment-related side effects and mortality rates are associated with the procedure.

Therefore, it's often considered safer to use a conservative therapy that doesn't involve transplantation for patients in low-risk or intermediate-1 risk groups. This approach has been shown to result in a lower risk of death compared to those who undergo transplantation. As a result, it's common for intermediate-2 and high-risk patients to receive allogeneic hematopoietic stem cell transplantation as a treatment. However, even in the intermediate-1 risk group, transplantation may be considered if patients require frequent transfusions, if immature cells in peripheral blood exceed 2%, or if there's no mutation in the JAK2/Calreticulin/MPL genes. Transplantation may also be considered if mutations of the ASXL1, EZH2, XRXF2, IDH1, or IDH2 genes are found in next-generation DNA sequencing, as these mutations can indicate a poor prognosis. For the remaining patients, it may be a reasonable alternative to delay transplantation until poor prognostic factors appear or resistance to existing treatment is shown.

To prepare for transplantation, pre-treatment (whole-body radiation therapy + high-dose anticancer chemotherapy) is performed to reduce fibrotic cells 51% and a one-year treatment-related mortality rate of 16%. It showed good treatment results compared to the prognosis of high-risk patients.

Summarizing the transplantation results of the Catholic Hematology Hospital, the two-year disease-free survival rate (the probability of survival without relapse and suppress cells rejecting the donor cells. When transplantation is performed with bone marrow-ablative pre-treatment, the overall 5-year survival rate of myelofibrosis and death from transplantation side effects) reached about 60~70%. The probability of death due to transplantation side effects was about is 30-40%, and disease-free survival rate is 22-33%, with significant morbidity and mortality, making it difficult to predict the benefits of transplantation 20 ~ 25%, and the mortality rate due to recurrence or progression of the disease was about 510%. Transplantation results showed better results in genetically.

These days, low-dose pre-treatment transplantation, which reduces the intensity of anticancer drugs or radiation, is mainly performed, making transplantation perfectly matched siblings or unrelated donors than in genetically mismatched or half-matched family transplants.