Gagelmann N, Badbaran A, Salit RB, et al. Impact of TP53 on Outcome of Patients with Myelofibrosis Undergoing Hematopoietic Stem Cell Transplantation. Blood. 2023; (doi: 10.1182/blood.2023019630).
For patients with myelofibrosis undergoing hematopoietic stem cell transplantation, multi-hit TP53 mutations (TP53MT) are a very high risk group, while the outcomes of those developing single-hit TP53MT were similar to those without mutations, new research shows. The study included 349 patients, with TP53MT detected in 49, including 30 who had a multi-hit configuration. The median variant allele frequency was 20.3%, while cytogenic risk was favorable for 71%, unfavorable for 23%, and very high for 6%. Thirty-six patients (10%) had complex karyotypes. TP53MT patients had a 1.5-year median survival rate compared with 13.5 years for patients with TP53 wild-type (TP53WT). Six-year survival for multi-hit TP53MT patients was 25%, compared with 56% for single-hit TP53MT patients and 64% for TP53WT carriers. Transplant-specific risk factors and conditioning intensity did not affect outcomes. Single-hit TP53MT patients had a 17% cumulative incidence of relapse vs 52% for multi-hit TP53MT patients and 21% for TP53WT. Ten TP53MT patients had leukemic transformation soon after transplantation compared with seven TP53WT patients. Eight of 10 patients with TP53MT had a multi-hit configuration. The median time to leukemic transformation for multi-hit TP53MT, single-hit TP53MT, and TP53WT was 0.7 years, 0.5 years, and 2.5 years, respectively.
Tags: hematology, Research, Science, abstract, mutation, results, hematopoietic transplantation