2,285
Views
0
CrossRef citations to date
0
Altmetric
Research Article

European Delphi panel to build consensus on tapering and discontinuing thrombopoietin receptor agonists in immune thrombocytopenia

, , &
Article: 2170999 | Received 15 Nov 2022, Accepted 17 Jan 2023, Published online: 21 Feb 2023

References

  • Kistangari G, McCrae KR. Immune thrombocytopenia. Hematol Oncol Clin North Am. 2013;27(3):495–8. doi:10.1016/j.hoc.2013.03.001.
  • Kohli R, Chaturvedi S. Epidemiology and clinical manifestations of immune thrombocytopenia. Hämostaseologie. 2019;39(3):238–249. doi:10.1055/s-0039-1683416.
  • Trotter P, Hill QA. Immune thrombocytopenia: improving quality of life and patient outcomes. Pat Relat Outcome Meas. 2018;9:369. doi:10.2147/PROM.S140932.
  • European Medicines Agency. Guideline on the clinical development of medicinal products intended for the treatment of chronic primary immune thrombocytopenia. Oncology Working Party, editor. London, UK; 2014.
  • Moulis G, Palmaro A, Montastruc J-L, Godeau B, Lapeyre-Mestre M, Sailler L. Epidemiology of incident immune thrombocytopenia: a nationwide population-based study in France. Blood. 2014;124(22):3308–3315. doi:10.1182/blood-2014-05-578336.
  • Lambert MP, Gernsheimer TB. Clinical updates in adult immune thrombocytopenia. Blood. 2017;129(21):2829–2835. doi:10.1182/blood-2017-03-754119.
  • Cooper N, Hill QA, Grainger J, Westwood J-P, Bradbury C, Provan D, Thachil J, Ramscar N, Roy A. Tapering and discontinuation of thrombopoietin receptor agonist therapy in patients with immune thrombocytopenia: results from a modified Delphi panel. Acta Haematol. 2021;144(4):418–426. doi:10.1159/000510676.
  • Bussel JB, Mahmud SN, Brigstocke SL, Torneten SM. Tapering eltrombopag in patients with chronic ITP: how successful is this and in whom does it work? Blood. 2015;126(23):1054. doi:10.1182/blood.V126.23.1054.1054.
  • Khellaf M, Michel M, Quittet P, Viallard J-F, Alexis M, Roudot-Thoraval F, Cheze S, Durand J-M, Lefrère F, Galicier L, et al. Romiplostim safety and efficacy for immune thrombocytopenia in clinical practice: 2-year results of 72 adults in a romiplostim compassionate-use program. Blood. 2011;118(16):4338–4345. doi:10.1182/blood-2011-03-340166.
  • Saleh MN, Bussel JB, Cheng G, Meyer O, Bailey CK, Arning M, Brainsky A. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study. Blood. 2013;121(3):537–545. doi:10.1182/blood-2012-04-425512.
  • Leven E, Miller A, Boulad N, Haider A, Bussel JB. Successful discontinuation of eltrombopag treatment in patients with chronic ITP. Blood. 2012;120(21):1085. doi:10.1182/blood.V120.21.1085.1085.
  • Rodeghiero F, Carli G. Beyond immune thrombocytopenia: the evolving role of thrombopoietin receptor agonists. Ann Hematol. 2017;96(9):1421–1434. doi:10.1007/s00277-017-2953-6.
  • Carpenedo M, Baldacci E, Baratè C, Borchiellini A, Buccisano F, Calvaruso G, Chiurazzi F, Fattizzo B, Giuffrida G, Rossi E, et al. Second-line administration of thrombopoietin receptor agonists in immune thrombocytopenia: Italian Delphi-based consensus recommendations. Ther Adv Hematol. 2021;12:20406207211048361. doi:10.1177/20406207211048361.
  • Cuker A, Despotovic JM, Grace RF, Kruse C, Lambert MP, Liebman HA, Lyons RM, McCrae KR, Pullarkat V, Wasser JS, et al. Tapering thrombopoietin receptor agonists in primary immune thrombocytopenia: expert consensus based on the RAND/UCLA modified Delphi panel method. Res Pract Thromb Haemost. 2021;5(1):69–80. doi:10.1002/rth2.12457.
  • Dalkey N, Helmer O. An experimental application of the Delphi method to the use of experts. Manage Sci. 1963;9(3):458–467. doi:10.1287/mnsc.9.3.458.
  • Fink A, Kosecoff J, Chassin M, Brook RH. Consensus methods: characteristics and guidelines for use. Am J Public Health. 1984;74(9):979–983. doi:10.2105/AJPH.74.9.979.
  • Rowe G, Wright G. Expert opinions in forecasting: the role of the Delphi technique. In: Scott Armstrong J, editor. Principles of forecasting. New York: Springer New York; 2001. p. 125–144.
  • Furniss D, Randell R, O’Kane AA, Taneva S, Mentis H, Blandford A. Fieldwork for healthcare: guidance for investigating human factors in computing systems. Synth Lect Assistive Rehabil Health Preserving Technol. 2014;3(2):1–146.
  • González-López TJ, Sánchez-González B, Pascual C, Arefi M, de Cabo E, Alonso A, Martín-Salces M, Jiménez-Bárcenas R, Calbacho M, Galan P, et al. Sustained response after discontinuation of short- and medium-term treatment with eltrombopag in patients with immune thrombocytopenia. Platelets. 2015;26(1):83–86. doi:10.3109/09537104.2013.870987.
  • Rashidi A, Blinder M. Combination therapy in relapsed or refractory chronic immune thrombocytopenia: a case report and literature review. J Clin Pharm Therap. 2016;41(5):453–458. doi:10.1111/jcpt.12421.
  • Ghadaki B, Nazi I, Kelton JG, Arnold DM. Sustained remissions of immune thrombocytopenia associated with the use of thrombopoietin receptor agonists. Transfusion. 2013;53(11):2807–2812. doi:10.1111/trf.12139.
  • Iino M, Sakamoto Y, Sato T. Treatment-free remission after thrombopoietin receptor agonist discontinuation in patients with newly diagnosed immune thrombocytopenia: an observational retrospective analysis in real-world clinical practice. Int J Hematol. 2020;112(2):159–168. doi:10.1007/s12185-020-02893-y.
  • González‐López TJ, Pascual C, Álvarez‐román MT, Fernández-Fuertes F, Sánchez-González B, Caparrós I, Jarque I, Mingot-Castellano ME, Hernández-Rivas JA, Martín-Salces M, et al. Successful discontinuation of eltrombopag after complete remission in patients with primary immune thrombocytopenia. Am J Hematol. 2015;90(3):E40–43. doi:10.1002/ajh.23900.
  • Červinek L, Mayer J, Doubek M. Sustained remission of chronic immune thrombocytopenia after discontinuation of treatment with thrombopoietin-receptor agonists in adults. Int J Hematol. 2015;102(1):7–11. doi:10.1007/s12185-015-1793-1.
  • Zhang L, Zhang M, Du X, Cheng Y, Cheng G. Safety and efficacy of eltrombopag plus pulsed dexamethasone as first‐line therapy for immune thrombocytopenia. Brit J Haematol. 2020;189(2):369–378. doi:10.1111/bjh.16327.
  • Wong RS, Saleh MN, Khelif A, Salama A, Portella MSO, Burgess P, Bussel JB. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. 2017;130(23):2527–2536. doi:10.1182/blood-2017-04-748707.
  • Zaja F, Carpenedo M, Baratè C, Borchiellini A, Chiurazzi F, Finazzi G, Lucchesi A, Palandri F, Ricco A, Santoro C, et al. Tapering and discontinuation of thrombopoietin receptor agonists in immune thrombocytopenia: real-world recommendations. Blood Rev. 2020;41:100647. doi:10.1016/j.blre.2019.100647.
  • Provan D, Arnold DM, Bussel JB, Chong BH, Cooper N, Gernsheimer T, Ghanima W, Godeau B, González-López TJ, Grainger J, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv. 2019;3(22):3780–3817. doi:10.1182/bloodadvances.2019000812.
  • Taylor E. We agree, don’t we? The Delphi method for health environments research. HERD. 2020;13(1):11–23. doi:10.1177/1937586719887709.
  • Gonzalez-Lopez TJ, Fernandez-Fuertes F, Izquierdo MCP, Caparros I, Bernat S, Pastoriza C, Martínez-Badas MP, Olivera PE, Caballero G, Hernandez-Rivas JA, et al. Long term discontinuation of eltrombopag after remission in primary immune thrombocytopenia: 8-year follow-up data from 15 Spanish centers. Blood. 2019;134(Supplement_1):2352. doi:10.1182/blood-2019-129274.
  • Fowler FJ Jr, Levin CA, Sepucha KR. Informing and involving patients to improve the quality of medical decisions. Health Aff. 2011;30(4):699–706. doi:10.1377/hlthaff.2011.0003.
  • Hawley ST, Morris AM. Cultural challenges to engaging patients in shared decision making. Patient Educ Counsel. 2017;100(1):18–24. doi:10.1016/j.pec.2016.07.008.
  • Song F, Al-Samkari H. Management of adult patients with immune thrombocytopenia (ITP): a review on current guidance and experience from clinical practice. J Blood Med. 2021;12:653. doi:10.2147/JBM.S259101.
  • Thachil J, Bagot C, Bradbury C, Cooper N, Lester W, Grainger JD, Lowe G, Evans G, Talks K, Sibson K, et al. A United Kingdom Immune Thrombocytopenia (ITP) forum review of practice: thrombopoietin receptor agonists. Brit J Haematol. 2018;180(4):591–594. doi:10.1111/bjh.14395.
  • Mahevas M, Guillet S, Viallard J-F, Gobert D, Malphettes M, Cheze S, Lefrere F, Audia S, Bonnotte B, Lambotte O, et al. Rate of prolonged response after stopping thrombopoietin-receptor agonists treatment in primary immune thrombocytopenia (ITP): results from a nationwide prospective multicenter interventional study (STOPAGO). Blood. 2021;138(Supplement 1):583. doi:10.1182/blood-2021-152767.
  • Mahévas M, Fain O, Ebbo M, Roudot-Thoraval F, Limal N, Khellaf M, Schleinitz N, Bierling P, Languille L, Godeau B, et al. The temporary use of thrombopoietin‐receptor agonists may induce a prolonged remission in adult chronic immune thrombocytopenia. Results of a French observational study. Brit J Haematol. 2014;165(6):865–869. doi:10.1111/bjh.12888.
  • Lucchini E, Palandri F, Volpetti S, Vianelli N, Auteri G, Rossi E, Patriarca A, Carli G, Barcellini W, Tosi P, et al. Eltrombopag as second line therapy in adult patients with primary immune thrombocytopenia (ITP) in attempt to achieve long-term remission. preliminary analysis of a phase ii, multicenter, prospective study by gimema group (the ESTIT study). Blood. 2018;132(Supplement 1):1135. doi:10.1182/blood-2018-99-113314.
  • Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009;113(11):2386–2393. doi:10.1182/blood-2008-07-162503.
  • Vogel C, Zwolinsky S, Griffiths C, Hobbs M, Henderson E, Wilkins E. A Delphi study to build consensus on the definition and use of big data in obesity research. Int J Obesity. 2019 Dec 1;43(12):2573–2586. doi:10.1038/s41366-018-0313-9.