Real-life use of ropeg-interferon α2b in polycythemia vera: patient selection and clinical outcomes.
Cytoreduction
Myeloproliferative neoplasms
Polycythemia vera
Real-life
Ropeg-interferon α2b
Journal
Annals of hematology
ISSN: 1432-0584
Titre abrégé: Ann Hematol
Pays: Germany
ID NLM: 9107334
Informations de publication
Date de publication:
21 May 2024
21 May 2024
Historique:
received:
20
03
2024
accepted:
14
05
2024
medline:
21
5
2024
pubmed:
21
5
2024
entrez:
21
5
2024
Statut:
aheadofprint
Résumé
Ropeginterferon-alfa2b (ropegIFNα2b) is a long-acting IFN formulation with broad FDA/EMA approval as a therapy of polycythemia vera (PV) with no symptomatic splenomegaly. There is currently lack of information on the real-world patient selection, including the impact of local reimbursement policies, and drug management, particularly: type/timing of screening and follow-up tests; absolute/relative contraindications to therapy; ropegIFNα2b dose and combinations with hydroxyurea. As a sub-analysis of the PV-ARC retrospective study (NCT06134102), we here report our monocenter experience with ropegIFNα2b in the period from January 2021, corresponding to drug availability outside clinical trial, and December 2023. Among the 149 patients with EMA/FDA indication, only 55 (36.9%) met the local reimbursement criteria and 18 (12.1%) received ropegIFNα2b. Thanks to appropriate screening, relative/absolute contraindications to ropegIFNα2b were detected and managed in a multidisciplinary manner. Efficacy and safety of ropegIFNα2b was confirmed, with 3 cases of early molecular response. General use of low ropegIFNα2b dose, with frequent need for hydroxyurea combinations, was noted. This real-world experience suggests a significant impact of local regulations on drug prescription and the need for greater real-world data collection on ropegIFNα2b in PV patients. Also, it describes appropriate multidisciplinary screening and monitoring procedures during ropegIFNα2b therapy.
Identifiants
pubmed: 38771499
doi: 10.1007/s00277-024-05809-6
pii: 10.1007/s00277-024-05809-6
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Informations de copyright
© 2024. The Author(s).
Références
Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM et al (2016) The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood 127(20):2391–2405
doi: 10.1182/blood-2016-03-643544
pubmed: 27069254
Marchetti M, Vannucchi AM, Griesshammer M, Harrison C, Koschmieder S, Gisslinger H et al (2022) Appropriate management of polycythaemia vera with cytoreductive drug therapy: European LeukemiaNet 2021 recommendations. Lancet Haematol 9(4):e301–e311
doi: 10.1016/S2352-3026(22)00046-1
pubmed: 35358444
Tefferi A, Barbui T (2023) Polycythemia vera: 2024 update on diagnosis, risk-stratification, and management. Am J Hematol 98(9):1465–1487
doi: 10.1002/ajh.27002
pubmed: 37357958
Kiladjian JJ, Klade C, Georgiev P, Krochmalczyk D, Gercheva-Kyuchukova L, Egyed M et al (2022) Long-term outcomes of polycythemia vera patients treated with ropeginterferon Alfa-2b. Leukemia 36(5):1408–1411
doi: 10.1038/s41375-022-01528-x
pubmed: 35210530
pmcid: 9061291
Gisslinger H, Klade C, Georgiev P, Krochmalczyk D, Gercheva-Kyuchukova L, Egyed M et al (2023) Event-free survival in patients with polycythemia vera treated with ropeginterferon alfa-2b versus best available treatment. Leuk 2023 37(10):10
Barbui T, Vannucchi AM, De Stefano V, Masciulli A, Carobbio A, Ferrari A et al (2021) Ropeginterferon alfa-2b versus phlebotomy in low-risk patients with polycythaemia vera (Low-PV study): a multicentre, randomised phase 2 trial. Lancet Haematol 8(3):e175–e184
doi: 10.1016/S2352-3026(20)30373-2
pubmed: 33476571
Crisà E, Cerrano M, Beggiato E, Benevolo G, Lanzarone G, Manzini PM et al (2017) Can pegylated interferon improve the outcome of polycythemia vera patients? J Hematol Oncol. ;10(1)
Bewersdorf JP, Giri S, Wang R, Podoltsev N, Williams RT, Tallman MS et al (2021) Interferon alpha therapy in essential thrombocythemia and polycythemia vera-a systematic review and meta-analysis. Leukemia 35(6):1643–1660
doi: 10.1038/s41375-020-01020-4
pubmed: 32868875
Palandri F, Rossi E, Auteri G, Breccia M, Paglia S, Benevolo G et al (2023) Predictors of response to Hydroxyurea and switch to Ruxolitinib in HU-Resistant Polycythaemia VERA patients: a real-world PV-NET study. Cancers (Basel). ;15(14)
Arber DA, Orazi A, Hasserjian RP, Borowitz MJ, Calvo KR, Kvasnicka HM et al (2022) International Consensus classification of myeloid neoplasms and Acute Leukemias: integrating morphologic, clinical, and genomic data. Blood 140(11):1200–1228
doi: 10.1182/blood.2022015850
pubmed: 35767897
pmcid: 9479031
Barosi G, Mesa R, Finazzi G, Harrison C, Kiladjian JJ, Lengfelder E et al (2013) Revised response criteria for polycythemia vera and essential thrombocythemia: an ELN and IWG-MRT consensus project. Blood 121(23):4778–4781
doi: 10.1182/blood-2013-01-478891
pubmed: 23591792
pmcid: 3674675
Jovanovic JV, Ivey A, Vannucchi AM, Lippert E, Oppliger Leibundgut E, Cassinat B et al (2013) Establishing optimal quantitative-polymerase chain reaction assays for routine diagnosis and tracking of minimal residual disease in JAK2-V617F-associated myeloproliferative neoplasms: a joint European LeukemiaNet/MPN&MPNr-EuroNet (COST action BM0902) study. Leukemia 27(10):2032
doi: 10.1038/leu.2013.219
pubmed: 23860450
pmcid: 3806250
Aguilera-Diaz A, Vazquez I, Ariceta B, Mañú A, Blasco-Iturri Z, Palomino-Echeverría S et al (2020) Assessment of the clinical utility of four NGS panels in myeloid malignancies. Suggestions for NGS panel choice or design. PLoS ONE. ;15(1)
Zigmond AS, Snaith RP (1983) The Hospital anxiety and Depression Scale. Acta Psychiatr Scand 67(6):361–370
doi: 10.1111/j.1600-0447.1983.tb09716.x
pubmed: 6880820
Zambrano-Román M, Padilla-Gutiérrez JR, Valle Y, Muñoz-Valle JF, Valdés-Alvarado E (2022) Non-Melanoma Skin Cancer: A Genetic Update and Future Perspectives. Cancers 2022, Vol 14, Page 2371. ;14(10):2371
AIFA. Determina 245/2022. 2022. Available from: https://www.google.com/url?sa=t&rct=j&q=&esrc=s&source=web&cd=&ved=2ahUKEwi0uM3h-eaDAxVMQvEDHVtpBKkQFnoECBEQAQ&url=https%3A%2F%2Fwww.aifa.gov.it%2Fdocuments%2F20142%2F961234%2FDetermina_245-2022_Besremi.pdf&usg=AOvVaw0DSegUKc2kY2DJyx_A6yMN&opi=89978449
Mora B, Passamonti F (2023) SOHO State of the art updates and next questions | Polycythemia Vera: is it time to Rethink Treatment? Clin Lymphoma Myeloma Leuk 23(2):79–85
doi: 10.1016/j.clml.2022.11.011
pubmed: 36566109
ropeginterferon alfa-2b Prescribing Information Available from: moz-extension://7ee338ac-f6ec-468b-8b49-f6638b2d6088/enhanced-reader.html?openApp&pdf = https%3A%2F%2Fwww.accessdata.fda.gov%2Fdrugsatfda_docs%2Flabel%2F2021%2F761166s000lbl.pdf
Jin J, Zhang L, Qin A, Wu D, Shao Z, Bai J et al (2023) A new dosing regimen of ropeginterferon alfa-2b is highly effective and tolerable: findings from a phase 2 study in Chinese patients with polycythemia vera. Exp Hematol Oncol 12(1):1–5
doi: 10.1186/s40164-023-00415-0
Balderramo DC, García O, Colmenero J, Espinosa G, Forns X, Ginès P (2009) Antiphospholipid syndrome during pegylated interferon alpha-2a therapy for chronic hepatitis C. Dig Liver Dis 41(7):e4
doi: 10.1016/j.dld.2007.11.029
pubmed: 18243078
Hubben A, McCrae KR (2023) How to diagnose and manage antiphospholipid syndrome. Hematol Am Soc Hematol Educ Program 2023(1):606–613
doi: 10.1182/hematology.2023000493
Xourgia E, Tektonidou MG (2019) Type I interferon gene expression in antiphospholipid syndrome: pathogenetic, clinical and therapeutic implications. Journal of Autoimmunity, vol 104. Academic
Verger E, Cassinat B, Aur´ A, Chauveau A, Dosquet C, Giraudier S et al (2015) Clinical and molecular response to interferon-a therapy in essential thrombocythemia patients with CALR mutations
Quintás A, Quintás-Cardama Q, Abdel-Wahab O, Manshouri T, Kilpivaara O, Cortes J et al (2013) Molecular analysis of patients with polycythemia vera or essential thrombocythemia receiving pegylated interferon a-2a
Kiladjian JJ, Massé A, Cassinat B, Mokrani H, Teyssandier I, Le Couédic JP et al (2010) Clonal analysis of erythroid progenitors suggests that pegylated interferon alpha-2a treatment targets JAK2V617F clones without affecting TET2 mutant cells. Leukemia 24(8):1519–1523
doi: 10.1038/leu.2010.120
pubmed: 20520643
Ciążyńska M, Kamińska-Winciorek G, Lange D, Lewandowski B, Reich A, Sławińska M et al (2021) The incidence and clinical analysis of non-melanoma skin cancer. Sci Rep 2021 11(1):1
Polverelli N, Elli EM, Abruzzese E, Palumbo GA, Benevolo G, Tiribelli M et al (2021) Second primary malignancy in myelofibrosis patients treated with ruxolitinib. Br J Haematol 193(2):356–368
doi: 10.1111/bjh.17192
pubmed: 33222197
Barbui T, Ghirardi A, Masciulli A, Carobbio A, Palandri F, Vianelli N et al (2019) Second cancer in Philadelphia negative myeloproliferative neoplasms (MPN-K). A nested case-control study. Leukemia 33(8):1996–2005
doi: 10.1038/s41375-019-0487-8
pubmed: 31142846
Grigg A (2007) Effect of hydroxyurea on sperm count, motility and morphology in adult men with sickle cell or myeloproliferative disease. Intern Med J 37(3):190–192
doi: 10.1111/j.1445-5994.2006.01290.x
pubmed: 17316339
Jinna S, Khandhar PB, Hydroxyurea Toxicity (2023) StatPearls. Aug 8
Barbui T, Carobbio A, De Stefano V, Alvarez-Larran A, Ghirardi A, Carioli G et al (2023) Ropeginterferon phase 2 randomized study in low-risk polycythemia vera: 5-year drug survival and efficacy outcomes. Ann Hematol
Beauverd Y, Ianotto JC, Thaw KH, Sobas M, Sadjadian P, Curto-Garcia N et al (2023) Impact of Cytoreductive drugs upon outcomes in a contemporary cohort of adolescent and young adults with essential thrombocythemia and Polycythemia Vera. Blood 142(Supplement 1):748–748
doi: 10.1182/blood-2023-185108