Blood Res.  2022 Mar;57(1):59-68. 10.5045/br.2021.2021209.

Myelofibrotic and leukemic transformation in 2016 WHO-defined Philadelphia-negative myeloproliferative neoplasm

• Song IC
• Yeon SH
• Lee MW
• Ryu H
• Lee HJ
• Yun HJ
• Kim SY
• Jo DY

• KMID: 2527498
• DOI: http://doi.org/10.5045/br.2021.2021209

Abstract

Background
Information on myelofibrotic and leukemic transformations in Korean Philadelphia chromosome-negative myeloproliferative neoplasms (Ph ‒ MPNs) is limited.
Methods
This study retrospectively analyzed transformations in patients diagnosed with essential thrombocythemia (ET), polycythemia vera (PV) prefibrotic/early primary myelofibrosis (pre-PMF), or overt primary myelofibrosis (PMF) based on the 2016 World Health Organization criteria between January 1996 and December 2020 at Chungam National University Hospital, Daejeon, Korea.
Results
A total of 351 patients (144 with ET, 131 with PV, 45 with pre-PMF, and 31 with PMF; 204 men and 147 women) with a median age of 64 years (range, 15‒91 years) were followed for a median of 4.6 years (range, 0.2‒24.8 years). The 10-year incidence of overt myelofibrosis was higher in pre-PMF than in ET (31.3% and 13.7%, respectively; P =0.031) and PV (12.2%; P =0.003). The 10-year incidence of leukemic transformation was significantly higher in PMF than in ET (40.0% and 7.9%, respectively; P =0.046), pre-PMF (4.7%; P =0.048), and PV (3.2%; P =0.031). The 5-year incidence of leukemic transformation was higher in patients with secondary myelofibrosis (SMF) than in those with PMF (19.0% and 11.4%, respectively; P =0.040). The 5-year overall survival of patients with SMF was significantly worse than that of patients with pre-PMF (74% and 93%, respectively; P =0.027) but did not differ from that of patients with PMF (57%; P =0.744).
Conclusion
The rates and clinical courses of myelofibrotic and leukemic transformations in Korean patients with Ph ‒ MPN did not differ from those in Western populations.

Keyword

Myeloproliferative neoplasm; Essential thrombocythemia; Polycythemia vera; Primary myelofibrosis; Secondary myelofibrosis; Leukemia

Figure

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  Fig. 1 Myelofibrotic and leukemic transformations in patients with myeloproliferative neoplasm. Abbreviations: AML, acute myeloid leukemia; ET, essential thrombo-cythemia; PMF, overt primary myelofibrosis; pre-PMF, prefibrotic/early primary myelofibrosis; PV, polycythemia vera.

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  Fig. 2 Cumulative incidence of myelofibrotic transformation in myeloproliferative neoplasms. Abbreviations: ET, essential thrombocythemia; pre-PMF, prefibrotic/early primary myelofibrosis; PV, polycythemia vera.

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  Fig. 3 Cumulative incidence of leukemic transformation in myelo-proliferative neoplasms. Abbreviations: ET, essential thrombocythemia; PMF, overt primary myelofibrosis; pre-PMF, prefibrotic/early primary myelofibrosis; PV, polycythemia vera; SMF, secondary myelofibrosis.

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  Fig. 4 Overall survival of secondary myelofibrosis, prefibrotic/early primary myelofibrosis, and overt primary myelofibrosis. Abbreviations: OS, overall survival; PMF, overt primary myelofibrosis; pre-PMF, prefibrotic/early primary myelofibrosis; SMF, secondary myelofibrosis.

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