Scholarlink

<p>Patients with blast-phase (BP) myeloproliferative neoplasms have dismal outcomes, but allogeneic hematopoietic stem cell transplantation (alloHSCT) may offer a potential cure. However, the optimal pre-transplant blast-reduction therapy remains to be determined. We retrospectively analyzed outcomes in a molecularly annotated cohort of 24 patients with BP myelofibrosis (MF) who underwent alloHSCT between 2008 and 2023. Before conditioning, 20 patients received intensive induction chemotherapy, 1 received decitabine plus venetoclax, and 3 proceeded directly to transplant. At transplantation, 13 patients (54%) were in complete remission (CR), defined as bone marrow blast &lt; 5%, or CR with incomplete count recovery (CRi), while 11 (46%) had active disease. The median follow-up was 2.9 years (range 0.02–15.5). The 3-year overall survival (OS) was 62%, progression-free survival (PFS) was 49%, relapse incidence was 38%, and non-relapse mortality was 12%. There was a trend toward better OS and PFS in patients transplanted in CR/CRi compared with those with active disease (OS 68% vs. 55%, HR 0.42, <i>p</i> = 0.16; PFS 61% vs. 36%, HR 0.49, <i>p</i> = 0.21). Mutations in <i>TP53</i> and <i>EZH2</i> were associated with significantly inferior PFS (HR 6.28, <i>p</i> = 0.008 for <i>TP53</i> and HR 3.8, <i>p</i> = 0.04 for <i>EZH2</i>).</p><p>Compared with a historical cohort of 50 patients transplanted during the same period for chronic-phase MF, outcomes were similar (3-year OS: 62% vs. 58%; <i>p =</i> 0.6181). In conclusion, our results suggest that achieving remission before alloHSCT in BP-MF is associated with favorable outcomes. The adverse impact of <i>TP53</i> or <i>EZH2</i> mutations supports early post-transplant strategies to prevent relapse.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Outcomes of allogeneic hematopoietic stem cell transplantation in patients with blast phase myelofibrosis: molecular signature and intensive chemotherapy matter

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年5月30日

查看全文
  • Francesca Valsecchi,
  • Maria Chiara Finazzi,
  • Silvia Salmoiraghi,
  • Chiara Pavoni,
  • Anna Grassi,
  • Alessandra Algarotti,
  • Marta Bellini,
  • Marco Frigeni,
  • Annalisa Condorelli,
  • Benedetta Rambaldi,
  • Giuliana Rizzuto,
  • Gianluca Cavallaro,
  • Clara Belotti,
  • Martina Milani,
  • Orietta Spinelli,
  • Federico Lussana,
  • Alessandro Rambaldi

摘要

Patients with blast-phase (BP) myeloproliferative neoplasms have dismal outcomes, but allogeneic hematopoietic stem cell transplantation (alloHSCT) may offer a potential cure. However, the optimal pre-transplant blast-reduction therapy remains to be determined. We retrospectively analyzed outcomes in a molecularly annotated cohort of 24 patients with BP myelofibrosis (MF) who underwent alloHSCT between 2008 and 2023. Before conditioning, 20 patients received intensive induction chemotherapy, 1 received decitabine plus venetoclax, and 3 proceeded directly to transplant. At transplantation, 13 patients (54%) were in complete remission (CR), defined as bone marrow blast < 5%, or CR with incomplete count recovery (CRi), while 11 (46%) had active disease. The median follow-up was 2.9 years (range 0.02–15.5). The 3-year overall survival (OS) was 62%, progression-free survival (PFS) was 49%, relapse incidence was 38%, and non-relapse mortality was 12%. There was a trend toward better OS and PFS in patients transplanted in CR/CRi compared with those with active disease (OS 68% vs. 55%, HR 0.42, p = 0.16; PFS 61% vs. 36%, HR 0.49, p = 0.21). Mutations in TP53 and EZH2 were associated with significantly inferior PFS (HR 6.28, p = 0.008 for TP53 and HR 3.8, p = 0.04 for EZH2).

Compared with a historical cohort of 50 patients transplanted during the same period for chronic-phase MF, outcomes were similar (3-year OS: 62% vs. 58%; p = 0.6181). In conclusion, our results suggest that achieving remission before alloHSCT in BP-MF is associated with favorable outcomes. The adverse impact of TP53 or EZH2 mutations supports early post-transplant strategies to prevent relapse.