Patient-reported treatment burden of chronic immune thrombocytopenia therapies

BMC Hematology

Chris L Pashos²  Axel C Matzdorff³  Kelly M Grotzinger⁴  Ruslan V Horblyuk⁴  T Michelle Brown¹ 

[1] RTI Health Solutions, Research Triangle Park, NC, USA;United BioSource Corporation, Lexington, MA, USA;Department of Hematology and Oncology, Caritasklinikum Saarbruecken, St. Theresia, Saarbruecken, Germany;GlaxoSmithKline, Collegeville, PA, USA

关键词: Corticosteroid;    Bother;    Burden;    ITP;    Immune thrombocytopenia;   

Others  :  865405
DOI  :  10.1186/1471-2326-12-2

 received in 2011-06-13, accepted in 2012-03-22,  发布年份 2012

【 摘 要 】

Background

Chronic immune thrombocytopenia (ITP) is a debilitating autoimmune disorder that causes a reduction in blood platelets and increased risk of bleeding. ITP is currently managed with various pharmacologic therapies and splenectomy.

This study was conducted to assess patient perceived and reported treatment side effects, as well as the perceived burden or bother, and need to reduce or stop treatment, associated with these side effects among adult patients with chronic ITP.

Methods

A Web-enabled survey was administered to members of a US-based ITP patient support group. Patients reported demographic and clinical characteristics, ITP treatments' side effects for treatments received since diagnosed, level of bother (or distress), and need to reduce or stop treatment, associated with side effects. Current and past exposure was assessed for five specific treatment types: corticosteroids (CS), intravenous immunoglobulin (IVIg), anti-D immunoglobulin (anti-D), rituximab (RT), and splenectomy (SPL), as well as for other patient-referenced therapies (captured as "other").

Results

The survey was completed by 589 patients; 78% female, 89% white, mean age 48 years (SD = 14.71), and 68% reported a typical low platelet count of < 50,000/μL. Current or past treatment with CS was reported by 92% (n = 542) of patients, 56% (n = 322) for IVIg, 36% (n = 209) for anti-D, 36% (n = 213) for RT, and 39% (n = 227) for SPL. A substantial proportion of CS-treated patients reported side effects (98%, P < 0.05), were highly bothered by their side effects (53.1%, P < 0.05), and reported the need to stop or reduce treatment due to side effects (37.8%, P < 0.05). Among patients reporting side effects of treatment, significant associations were noted for the number of side effects, aggregate bother of reported side effects, and the need to stop or reduce treatment (all P < 0.05).

Conclusions

Current ITP treatments, particularly corticosteroids, are associated with multiple bothersome side effects that may lead to patients stopping or reducing therapy. Open, informed and complete communication between clinician and patient regarding both the benefits and the side effects of ITP treatment may better prepare patients for their prescribed regimens.

【 授权许可】

   
2012 Brown et al; licensee BioMed Central Ltd.

【 图 表 】

【 参考文献 】

• [1]Cines DB, Blanchette VS: Immune thrombocytopenic purpura. N Engl J Med 2002, 346(13):995-1008.
• [2]McMillan R, Nugent D: The effect of antiplatelet autoantibodies on megakaryocytopoiesis. Int J Hematol 2005, 81(2):94-99.
• [3]McMillan R, Bussel JB, George JN, Lalla D, Nichol JL: Self-reported health-related quality of life in adults with chronic immune thrombocytopenic purpura. Am J Hematol 2008, 83(2):150-154.
• [4]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. J Thromb Haemost 2008, 6(4):711-712. author reply 713
• [5]Segal JB, Powe NR: Prevalence of immune thrombocytopenia: analyses of administrative data. J Thromb Haemost 2006, 4(11):2377-2383.
• [6]George JN: Management of patients with refractory immune thrombocytopenic purpura. J Thromb Haemost 2006, 4(8):1664-1672.
• [7]Cohen YC, Djulbegovic B, Shamai-Lubovitz O, Mozes B: The bleeding risk and natural history of idiopathic thrombocytopenic purpura in patients with persistent low platelet counts. Arch Intern Med 2000, 160(11):1630-1638.
• [8]Portielje JE, Westendorp RG, Kluin-Nelemans HC, Brand A: Morbidity and mortality in adults with idiopathic thrombocytopenic purpura. Blood 2001, 97(9):2549-2554.
• [9]British Committee for Standards in Haematology General Haematology Task Force: Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. Br J Haematol 2003, 120(4):574-596.
• [10]Godeau B, Chevret S, Varet B, Lefrere F, Zini JM, Bassompierre F, Cheze S, Legouffe E, Hulin C, Grange MJ, et al.: Intravenous immunoglobulin or high-dose methylprednisolone, with or without oral prednisone, for adults with untreated severe autoimmune thrombocytopenic purpura: a randomised, multicentre trial. Lancet 2002, 359(9300):23-29.
• [11]Cines DB, Bussel JB: How I treat idiopathic thrombocytopenic purpura (ITP). Blood 2005, 106(7):2244-2251.
• [12]Kojouri K, Vesely SK, Terrell DR, George JN: Splenectomy for adult patients with idiopathic thrombocytopenic purpura: a systematic review to assess long-term platelet count responses, prediction of response, and surgical complications. Blood 2004, 104(9):2623-2634.
• [13]Godeau B, Porcher R, Fain O, et al.: Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood 2008, 112:999-1004.
• [14]Stasi R, Pagano A, Stipa E, Amadori S: Rituximab chimeric anti-CD20 monoclonal antibody treatment for adults with chronic idiopathic thrombocytopenic purpura. Blood 2001, 98(4):952-957.
• [15]Panzer S: New therapeutic options for adult chronic immune thrombocytopenic purpura: a brief review. Vox Sang 2008, 94(1):1-5.
• [16]Aledort LM, Salama A, Kovaleva L, Robak T, Newland AC, Nugent DJ, Brenner B, Zenker O: Efficacy and safety of intravenous anti-D immunoglobulin (Rhophylac) in chronic immune thrombocytopenic purpura. Hematology 2007, 12(4):289-295.
• [17]Orbach H, Katz U, Sherer Y, Shoenfeld Y: Intravenous immunoglobulin: adverse effects and safe administration. Clin Rev Allergy Immunol 2005, 29(3):173-184.
• [18]Kumar S, Diehn FE, Gertz MA, Tefferi A: Splenectomy for immune thrombocytopenic purpura: long-term results and treatment of postsplenectomy relapses. Ann Hematol 2002, 81(6):312-319.
• [19]Arnold DM, Dentali F, Crowther MA, Meyer RM, Cook RJ, Sigouin C, Fraser GA, Lim W, Kelton JG: Systematic review: efficacy and safety of rituximab for adults with idiopathic thrombocytopenic purpura. Ann Intern Med 2007, 146(1):25-33.
• [20]Mathias SD, Bussel JB, George JN, McMillan R, Okano GJ, Nichol JL: A disease-specific measure of health-related quality of life for use in adults with immune thrombocytopenic purpura: its development and validation. Health Qual Life Outcomes 2007, 5:11. BioMed Central Full Text
• [21]Sarpatwari A, Watson S, Anderson H, Provan D, Newland A: Health-related lifestyle among adult and pediatric patients with idiopathic thrombocytopenic purpura in the United Kingdom. 50th American Society of Hematology Annual Meeting 2008, 112(11):3435.
• [22]Fadem SZ, Walker DR, Abbott G, Friedman AL, Goldman R, Sexton S, Buettner K, Robinson K, Peters TG: Satisfaction with renal replacement therapy and education: the american association of kidney patients survey. Clin J Am Soc Nephrol 2011, 6(3):605-612.
• [23]Brown TM, Pashos CL, Joshi AV, Lee WC: The perspective of patients with haemophilia with inhibitors and their care givers: preferences for treatment characteristics. Haemophilia 2011, 17(3):476-482.
• [24]Poulin Y, Papp KA, Wasel NR, Andrew R, Fraquelli E, Bernstein G, Chan D: A Canadian online survey to evaluate awareness and treatment satisfaction in individuals with moderate to severe plaque psoriasis. Int J Dermatol 2010, 49(12):1368-1375.
• [25]Gore JL, Gollapudi K, Bergman J, Kwan L, Krupski TL, Litwin MS: Correlates of bother following treatment for clinically localized prostate cancer. J Urol 2010, 184(4):1309-1315.
• [26]Likert R: A technique for the measurement of attitudes. Arch Psych 1932, 140:1-55.
• [27]Fries AF: Survey of non-traditional treatments of ITP preliminary results. The Platelet News 2002, 3(3):4-5.
• [28]Matzdorff A, Arnold G: Treatment of chronic immune thrombocytopenic purpura: the patients' perspective. Eur J Haematol 2007, 78(5):381-388.
• [29]Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, et al.: Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med 2007, 357(22):2237-2247.