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Myeloproliferative neoplasms (MPN)

Transplant advances and outcomes

Myeloproliferative neoplasms (MPN) are a group of rare disorders, including BCR-ABL-negative myeloproliferative neoplasms, polycythemia vera, and essential thrombocytosis, where diagnosis usually occurs at a young age with a prognosis that depends on disease progression and effectiveness of conservative treatments. MPNs have been treated with conservative strategies over the years, but autologous HCT is an option that can prolong survival. [1-3]

Recommended timing for transplant consultation


Including primary myelofibrosis (PMF) and essential thrombocythemia or polycythemia vera that has progressed to MF (secondary MF).

High-resolution HLA typing and referral to HCT consultation is recommended at diagnosis for all patients with:

  • DIPSS or DIPSS Plus Intermediate-1 (INT-1) or higher
  • MIPSS70/MIPSS 70 plus version 2.0 intermediate-risk or higher
  • Cytopenic subtype
  • Young age
High-risk features including high-risk mutations ( ASXL1, TP53), triple negative (lack of a driver mutation such as JAK2, MPL or CALR)

  • Patients failing JAK inhibitor therapy

  • HCT is recommended upfront for patients with:

  • DIPSS or DIPSS Plus Intermediate-2 (INT-2) and high-risk disease

  • MIPSS70/MIPSS 70 plus version 2.0 high-risk disease

  • Patients with DIPSS INT-1 or MIPSS70/MIPSS 70 plus version 2.0 intermediate-risk, cytopenic subtype, young age, high-risk features, including high-risk mutations (ASXL1, TP53), triple negative (lack of a driver mutation such as JAK2, MPL, or CALR) and those failing JAK inhibitor therapy should be considered for upfront HCT balancing patient preferences and clinical trial options

References

  1. Tefferi A. How I treat myelofibrosis. Blood. 2011; 117(13): 3494-3504. Access
  2. Kröger NM, et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015; 29(11): 2126-2133. Access
  3. Deeg HJ, et al. Hematopoietic cell transplantation as curative therapy for patients with myelofibrosis: Long-term success in all age groups. Biol Blood Marrow Transplant. 2015; 21(11): 1883-1887. Access
  4. NMDP and ASTCT Recommended Timing for Transplant Consultation. Download PDF 
  5. National Comprehensive Cancer Network. Myeloproliferative Neoplasms. (Version 3.2022). Access