Ruxolitinib and interferon-alpha 2 combination therapy for patients with polycythemia vera or myelofibrosis: a phase II study
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Ruxolitinib and interferon-alpha 2 combination therapy for patients with polycythemia vera or myelofibrosis : a phase II study. / Sorensen, Anders Lindholm; Mikkelsen, Stine Ulrik; Knudsen, Trine Alma; Bjorn, Mads Emil; Andersen, Christen Lykkegaard; Bjerrum, Ole Weis; Brochmann, Nana; Patel, Dustin Andersen; Gjerdrum, Lise Mette Rahbek; El Fassi, Daniel; Kruse, Torben A.; Larsen, Thomas Stauffer; Mourits-Andersen, Hans Torben; Nielsen, Claus Henrik; Ellervik, Christina; Pallisgaard, Niels; Thomassen, Mads; Kjaer, Lasse; Skov, Vibe; Hasselbach, Hans Carl.
I: Haematologica, Bind 105, Nr. 9, 2020, s. 2262-2272.Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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TY - JOUR
T1 - Ruxolitinib and interferon-alpha 2 combination therapy for patients with polycythemia vera or myelofibrosis
T2 - a phase II study
AU - Sorensen, Anders Lindholm
AU - Mikkelsen, Stine Ulrik
AU - Knudsen, Trine Alma
AU - Bjorn, Mads Emil
AU - Andersen, Christen Lykkegaard
AU - Bjerrum, Ole Weis
AU - Brochmann, Nana
AU - Patel, Dustin Andersen
AU - Gjerdrum, Lise Mette Rahbek
AU - El Fassi, Daniel
AU - Kruse, Torben A.
AU - Larsen, Thomas Stauffer
AU - Mourits-Andersen, Hans Torben
AU - Nielsen, Claus Henrik
AU - Ellervik, Christina
AU - Pallisgaard, Niels
AU - Thomassen, Mads
AU - Kjaer, Lasse
AU - Skov, Vibe
AU - Hasselbach, Hans Carl
PY - 2020
Y1 - 2020
N2 - We report the final 2-year end-of-study results from the first clinical trial investigating combination treatment with ruxolitinib and low-dose pegylated interferon-alpha 2 (PEG-IFN alpha 2). The study included 32 patients with polycythemia vera and 18 with primary or secondary myelofibrosis; 46 patients were previously intolerant of or refractory to PEG-IFN alpha 2. The primary outcome was efficacy, based on hematologic parameters, quality of life measurements, and JAK2 V617F allele burden. We used the 2013 European LeukemiaNet and International Working Group-Myeloproliferative Neoplasms Research and Treatment response criteria, including response in symptoms, splenomegaly, peripheral blood counts, and bone marrow. Of 32 patients with polycythemia vera, ten (31%) achieved a remission which was a complete remission in three (9%) cases. Of 18 patients with myelofibrosis, eight (44%) achieved a remission; five (28%) were complete remissions. The cumulative incidence of peripheral blood count remission was 0.85 and 0.75 for patients with polycythemia vera and myelofibrosis, respectively. The Myeloproliferative Neoplasm Symptom Assessment Form total symptom score decreased from 22 [95% confidence interval (95% CI):, 16-29] at baseline to 15 (95% CI: 10-22) after 2 years. The median JAK2 V617F allele burden decreased from 47% (95% CI: 33-61%) to 12% (95% CI: 6-22%), and 41% of patients achieved a molecular response. The drop-out rate was 6% among patients with polycythemia vera and 32% among those with myelofibrosis. Of 36 patients previously intolerant of PEG-IFN alpha 2, 31 (86%) completed the study, and 24 (67%) of these received PEG-IFN alpha 2 throughout the study. In conclusion, combination treatment improved cell counts, reduced bone marrow cellularity and fibrosis, decreased JAK2 V617F burden, and reduced symptom burden with acceptable toxicity in several patients with polycythemia vera or myelofibrosis. #EudraCT2013-003295-12.
AB - We report the final 2-year end-of-study results from the first clinical trial investigating combination treatment with ruxolitinib and low-dose pegylated interferon-alpha 2 (PEG-IFN alpha 2). The study included 32 patients with polycythemia vera and 18 with primary or secondary myelofibrosis; 46 patients were previously intolerant of or refractory to PEG-IFN alpha 2. The primary outcome was efficacy, based on hematologic parameters, quality of life measurements, and JAK2 V617F allele burden. We used the 2013 European LeukemiaNet and International Working Group-Myeloproliferative Neoplasms Research and Treatment response criteria, including response in symptoms, splenomegaly, peripheral blood counts, and bone marrow. Of 32 patients with polycythemia vera, ten (31%) achieved a remission which was a complete remission in three (9%) cases. Of 18 patients with myelofibrosis, eight (44%) achieved a remission; five (28%) were complete remissions. The cumulative incidence of peripheral blood count remission was 0.85 and 0.75 for patients with polycythemia vera and myelofibrosis, respectively. The Myeloproliferative Neoplasm Symptom Assessment Form total symptom score decreased from 22 [95% confidence interval (95% CI):, 16-29] at baseline to 15 (95% CI: 10-22) after 2 years. The median JAK2 V617F allele burden decreased from 47% (95% CI: 33-61%) to 12% (95% CI: 6-22%), and 41% of patients achieved a molecular response. The drop-out rate was 6% among patients with polycythemia vera and 32% among those with myelofibrosis. Of 36 patients previously intolerant of PEG-IFN alpha 2, 31 (86%) completed the study, and 24 (67%) of these received PEG-IFN alpha 2 throughout the study. In conclusion, combination treatment improved cell counts, reduced bone marrow cellularity and fibrosis, decreased JAK2 V617F burden, and reduced symptom burden with acceptable toxicity in several patients with polycythemia vera or myelofibrosis. #EudraCT2013-003295-12.
KW - REVISED RESPONSE CRITERIA
KW - MINIMAL RESIDUAL DISEASE
KW - ESSENTIAL THROMBOCYTHEMIA
KW - MYELOPROLIFERATIVE NEOPLASMS
KW - ALLELE BURDEN
KW - OPEN-LABEL
KW - MOLECULAR RESPONSES
KW - LOW TOXICITY
KW - IWG-MRT
KW - ALPHA-2A
U2 - 10.3324/haematol.2019.235648
DO - 10.3324/haematol.2019.235648
M3 - Journal article
C2 - 33054051
VL - 105
SP - 2262
EP - 2272
JO - Haematologica
JF - Haematologica
SN - 0390-6078
IS - 9
ER -
ID: 249101466