References
- Cooper N, Ghanima W Immune Thrombocytopenia. Solomon CG. editor N Engl J Med. Internet 2019 Sep 5;381(10):945–11. doi: 10.1056/NEJMcp1810479
- Cines DB, Bussel JB, Liebman HA, et al. The ITP syndrome: pathogenic and clinical diversity. Blood Internet 2009 Jun 25;113(26):6511–6521. doi: 10.1182/blood-2009-01-129155
- Feudjo-Tepie MA, Robinson NJ, Bennett D. Prevalence of diagnosed chronic immune thrombocytopenic purpura in the US: analysis of a large US claim database: a rebuttal [5]. J Thromb Haemost. 2008;6(4):711–712. doi: 10.1111/j.1538-7836.2008.02911.x
- Rodeghiero F, Marranconi E. Management of immune thrombocytopenia in women: current standards and special considerations. Expert Rev Hematol. Internet 2020;13(2):175–185. doi: 10.1080/17474086.2020.1711729
- Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood Internet 2009 Mar 12;113(11):2386–2393. https://ashpublications.org/blood/article/113/11/2386/109971/Standardization-of-terminology-definitions-and
- Neunert C, Terrell DR, Arnold DM, et al. American society of hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019;3(23):3829–3866. doi: 10.1182/bloodadvances.2019000966
- Provan D, Arnold DM, Bussel JB, 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
- Despotovic JM, Grimes AB. Pediatric ITP: is it different from adult ITP? Hematology. Internet 2018 Nov 30;2018(1):405–411. doi: 10.1182/asheducation-2018.1.405
- Cooper N, Kruse A, Kruse C, et al. Immune thrombocytopenia (ITP) World Impact Survey (I-WISh): impact of ITP on health-related quality of life. Am J Hematol. Internet 2020 Feb 19;96(2):199–207. doi: 10.1002/ajh.26036
- Arnold DM. Bleeding complications in immune thrombocytopenia. Hematology. 2015 Dec 5;2015(1):237–242. Internet doi: 10.1182/asheducation-2015.1.237
- Matzdorff A, Meyer O, Ostermann H, et al. Immune thrombocytopenia - current diagnostics and therapy: recommendations of a joint working group of DGHO, ÖGHO, SGH, GPOH, and DGTI. Oncol Res Treat. 2018;41(Suppl 5):1–30. doi: 10.1159/000492187
- Provan D, Newland AC. Current management of primary immune thrombocytopenia. Adv Ther. 2015;32(10):875–887. doi: 10.1007/s12325-015-0251-z
- Neunert CE. Management of newly diagnosed immune thrombocytopenia: can we change outcomes? Blood Adv. 2017;1(24):2295–2301. doi: 10.1182/bloodadvances.2017009860
- Berti D, Moons P, Dobbels F, et al. Impact of corticosteroid-related symptoms in patients with immune thrombocytopenic purpura: results of a survey of 985 patients. Clin Ther. 2008;30(8):1540–1552. doi: 10.1016/j.clinthera.2008.08.005
- Brown TM, Horblyuk RV, Grotzinger KM, et al. Patient-reported treatment burden of chronic immune thrombocytopenia therapies. BMC Blood Disord. Internet 2012;12(1):2. doi: 10.1186/1471-2326-12-2
- Neunert C, Noroozi N, Norman G, et al. Severe bleeding events in adults and children with primary immune thrombocytopenia: a systematic review. J Thromb Haemost. Internet 2015 Mar;13(3):457–464. https://linkinghub.elsevier.com/retrieve/pii/S1538783622086627
- An R, Wang PP. Length of stay, hospitalization cost, and in-hospital mortality in US adult inpatients with immune thrombocytopenic purpura, 2006–2012. Vasc Health Risk Manag. 2017;13:15–21. doi: 10.2147/VHRM.S123631
- Agenzia Italiana del Farmaco. Determina n. 1060/2021. Available from: https://www.gazzettaufficiale.it/atto/serie_generale/caricaDettaglioAtto/originario?atto.dataPubblicazioneGazzetta=2021-09-21&atto.codiceRedazionale=21A05458&elenco30giorni=true.
- Agenzia Italiana del Farmaco. Legge 648/96. Lista faramaci ad uso consolidato. Available from: https://www.aifa.gov.it/legge-648-96
- Williams DD, Peng B, Bailey CK, et al. Effects of food and antacids on the pharmacokinetics of eltrombopag in healthy adult subjects: two single-dose, open-label, randomized-sequence, crossover studies. Clin Ther. 2009 Apr;31(4):764–776. https://linkinghub.elsevier.com/retrieve/pii/S0149291809001192
- Gilbert MM, Grimes AB, Kim TO, et al. Romiplostim for the treatment of immune thrombocytopenia: spotlight on patient acceptability and ease of use. Patient Prefer Adherence. 2020;14: 1237–1250. doi: 10.2147/PPA.S192481
- Bussel JB, Kuter DJ, Pullarkat V, et al. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. Internet 2009;113(10):2161–2171. doi: 10.1182/blood-2008-04-150078
- European Medicine Agency. REVOLADE. Summary of product characteristics. Available from: https://www.ema.europa.eu/en/documents/product-information/revolade-epar-product-information_en.pdf.
- European Medicine Agency. NPLATE. Summary of product characteristics. Available from: https://www.ema.europa.eu/en/documents/product-information/nplate-epar-product-information_en.pdf.
- Ghanima W, Junker P, Hasselbalch HC, et al. Fibroproliferative activity in patients with immune thrombocytopenia (ITP) treated with thrombopoietic agents. Br J Haematol. 2011;155(2):248–255. doi: 10.1111/j.1365-2141.2011.08845.x
- Kuter DJ, Mufti GJ, Bain BJ, et al. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. Internet 2009 Oct 29;114(18):3748–3756. https://ashpublications.org/blood/article/114/18/3748/26572/Evaluation-of-bone-marrow-reticulin-formation-in
- Jurczak W, Chojnowski K, Mayer J, et al. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018;183(3):479–490. doi: 10.1111/bjh.15573
- European Medicine Agency. DOPTELET. European assessment report. Available from: https://www.ema.europa.eu/en/documents/variation-report/doptelet-h-c-004722-ii-0004-g-epar-assessment-report-variation_en.pdf
- European Medicine Agency. DOPTELET. Summary of product characteristics. Available from: https://www.ema.europa.eu/en/documents/product-information/doptelet-epar-product-information_en.pdf
- Poordad F, Terrault NA, Alkhouri N, et al. Avatrombopag, an alternate treatment option to reduce platelet transfusions in patients with thrombocytopenia and chronic liver disease-integrated analyses of 2 phase 3 studies. Int J Hepatol. 2020;2020:1–11. doi: 10.1155/2020/5421632
- Agenzia Italiana del Farmaco. Determina n. 327/2022. Available from. https://www.gazzettaufficiale.it/atto/serie_generale/caricaDettaglioAtto/originario?atto.dataPubblicazioneGazzetta=2022-05-23&atto.codiceRedazionale=22A03007&elenco30giorni=true
- Sullivan SD, Mauskopf JA, Augustovski F, et al. Budget impact analysis—Principles of good practice: report of the ISPOR 2012 budget impact analysis good practice II task force. Value Heal. Internet 2014 Jan;17(1):5–14. https://linkinghub.elsevier.com/retrieve/pii/S1098301513042356
- Agenzia Italiana del Farmaco. Linee guida per la compilazione del dossier a supporto della domanda di rimborsabilità e prezzo. Available from: https://www.aifa.gov.it/documents/20142/1283800/Linee_guida_dossier_domanda_rimborsabilita.pdf
- Istituto Nazionale di Statistica (ISTAT). Previsioni della Popolazione Anni 2011-2065. Available from:http://demo.istat.it/. 2012. p. 22–25.
- Palandri F, Polverelli N, Sollazzo D, et al. Have splenectomy rate and main outcomes of ITP changed after the introduction of new treatments? A monocentric study in the outpatient setting during 35 years. Am J Hematol. 2016;91(4):E267–72. doi: 10.1002/ajh.24310
- Lambert MP, Gernsheimer TB. Clinical updates in adult immune thrombocytopenia. Blood. 2017;129(21):2829–2835. doi: 10.1182/blood-2017-03-754119
- IQVIA. Market research. Data on file.
- Kistangari G, McCrae KR. Immune thrombocytopenia. Hematol Oncol Clin North Am. 2013;27(3):495–520. doi: 10.1016/j.hoc.2013.03.001
- Ministero S. Decreto 18/10/2012 Remunerazione prestazioni di assistenza ospedaliera per acuti, assistenza ospedaliera di riabilitazione e di lungodegenza post acuzie e di assistenza specialistica ambulatoriale.
- Bussel J, Allen LF, Aggarwal K, et al. Lack of clinically significant hepatotoxicity in patients with chronic immune thrombocytopenia (c-ITP) treated with the novel, oral thrombopoietin receptor agonist avatrombopag-poole.
- Wong RSM, Saleh MN, Khelif A, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study. Blood. Internet 2017;130(23):2527–2536. doi: 10.1182/blood-2017-04-748707
- The National Institute for Health and Care Excellence. Eltrombopag for treating chronic immune (idiopathic) thrombocytopenic purpura - Technology appraisal guidance [Internet]. 2014 Jul. Available from: www.nice.org.uk/guidance/ta293
- Patient Access Monitor. Prezzi dei farmaci. Available from: https://pamonitor.it/LoginNew.aspx?ReturnUrl=%2fFarmaList.aspx
- Allen R, Bryden P, Grotzinger KM, et al. Cost-effectiveness of eltrombopag versus romiplostim for the treatment of chronic immune thrombocytopenia in England and wales. Value Heal. Internet 2016;19(5):614–622. doi: 10.1016/j.jval.2016.03.1856
- Tremblay G, Dolph M, Bhor M, et al. Cost-consequence model comparing eltrombopag versus romiplostim for adult patients with chronic immune thrombocytopenia. Clin Outcomes Res. Internet 2018;10:705–713.Nov. doi: 10.2147/CEOR.S177324
- Patwardhan P, Proudman D, Allen J, et al. Cost-minimization analysis comparing eltrombopag vs romiplostim for adults with chronic immune thrombocytopenia. J Manag Care Spec Pharm. 2021;27(10):1447–1456. doi: 10.18553/jmcp.2021.21080
- Fust K, Parthan A, Li X, et al. Cost per response analysis of strategies for chronic immune thrombocytopenia. Am J Manag Care. 2018;24:SP294–302.