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Table 1 Summary of clinical data for emerging/novel therapies for polycythemia vera

From: Emerging agents and regimens for polycythemia vera and essential thrombocythemia

Agent

Mechanism of action

Clinical data

Reference

Gandotinib

Pan-JAK inhibition

Phase 1 trial; N = 6 (median age 62.5 years)

Results: 3 patients had clinic-hematologic partial response [31]

Phase 2 trial; N = 20

Results: 4 patients (20%) had CR and 15 (75%) had PR, thus an ORR of 95% [32]

[31, 32]

Ruxolitinib + PEG-IFN alfa-2a

JAK1/2 inhibition + immunomodulation/antiangiogenesis

Phase 2 trial; N = 32 (median age 57 years, 66% high-risk, 94% with prior interferon exposure)

Results: 10 patients (31%) had remission with 3 (9%) in CR and another 7 (22%) with PR. Symptom burden was improved with only 6% drop-out rate.

[33]

Ropeginterferon alfa-2b

Immunomodulation/antiangiogenesis

Phase 3 trial; N = 222 (median age 58–60 years) randomized to ropeginterferon alfa-2b

Results: improved rates of composite CHR when compared with hydroxyurea (53% vs. 38%, p = 0.044) and improved symptom burden at 36 months follow-up

[34]

Givinostat

Histone deacetylase inhibition

Phase 1b/2 trial; N = 47

Results: ORR of 81%, but significant toxicity (35–50% grade ≤ 3 diarrhea, thrombocytopenia and creatinine elevation)

[35]

Idasanutlin

MDM2 inhibition

Phase 2 trial; N = 27 (median age 56 years, 26% with prior ruxolitinib exposure)

Results: 9 patients (56%) achieved hematocrit control by 32 weeks of therapy, however significant gastrointestinal toxicity

[36]

PTG-300

Hepcidin mimesis

Phase 2 trial; N = 13, all requiring therapeutic phlebotomy (mean age 57.4 years, 46% high-risk) Interim data showing normalization of iron stores, 100% therapeutic phlebotomy-independence and improved symptom burden

[37]

  1. Abbreviations: CHR complete hematological response, CR complete response, ORR overall response rate, PEG-IFN alfa-2a pegylated interferon alfa 2a, PR partial response