Immune thrombocytopenia: Effectiveness of frontline steroids and comparison of azathioprine, splenectomy, and rituximab as second‐line treatment

Chang, Hong-Yeh; Tang, Tzung‐Chih; Hung, Yu‐Shin; Li, Peiling; Kuo, Ming‐Chung; Wu, Jin‐Hou; Wang, Po‐Nan

doi:10.1111/ejh.13144

Cited by 19 publications

(15 citation statements)

References 46 publications

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“…Japanese health insurance). Azathioprine is reported to have a 40-60% efficacy rate with oral administration of 1-2 mg/kg/day (maximum dose 150 mg/day) [134,135]. Adverse drug reactions include myelosuppression, liver dysfunction, and pancreatitis.…”

Section: Azathioprine (Recommendation Level: 2c Not Covered Bymentioning

confidence: 99%

Reference guide for management of adult immune thrombocytopenia in Japan: 2019 Revision

et al. 2020

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Section: Azathioprine (Recommendation Level: 2c Not Covered Bymentioning

confidence: 99%

Reference guide for management of adult immune thrombocytopenia in Japan: 2019 Revision

et al. 2020

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“…Azathioprine dosed at 150mg/day has been reported to be effective in chronic refractory ITP [24,25]. One retrospective study spanning a six-month period demonstrated a response rate of 71.4%, with complete response in 38% of patients [24]. A prospective study of 53 patients showed a 64% response, with 40% maintaining a response after one year [25].…”

Section: Reviewmentioning

confidence: 99%

“…Another retrospective study of 96 patients found that 54% of patients responded to azathioprine, though only 2% had a sustained response [11]. Leukopenia and increases in transaminases were reported, as well as alopecia, gastrointestinal effects, and increased risk of malignancy, specifically lymphoma [24,25]. However, the sample sizes for many of these studies were small, so further studies are needed to solidify the evidence for the efficacy and safety of azathioprine.…”

Section: Reviewmentioning

confidence: 99%

Treatments for Primary Immune Thrombocytopenia: A Review

Samson¹,

Fraser²,

Lebowitz³

2019

Cureus

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Immune thrombocytopenic purpura (ITP) is an autoimmune condition that affects nearly 1:10,000 people in the world. It is traditionally defined by a platelet count of less than 100 x 109L, but treatment typically depends on symptomology rather than on the platelet count itself. For primary idiopathic ITP, corticosteroids have been the standard first-line of treatment for symptomatic patients, with the addition of intravenous immune globulin (IVIG) or Rho(D) immune globulin (anti-RhD) for steroid-resistant cases. In cases of refractory or non-responsive ITP, second-line therapy includes splenectomy or rituximab, a monoclonal antibody against the CD20 antigen (anti-CD20). In patients who continue to have severe thrombocytopenia and symptomatic bleeding despite first- and second-line treatments, the diagnosis of “chronic refractory ITP” is appropriate, and third-line treatments are evaluated. This manuscript describes the efficacy of different treatment options for primary ITP and introduces the reader to various third-line options that are emerging as a means of treating chronic refractory ITP.

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“…4,5 The authors obtain a surprisingly high rate of success and explain the discrepancy with former studies with "difference in case enrollment and definition of response". 1 The outcome of a high number of ITP cases has improved since the addition of rituximab and thrombopoietin receptor agonists to the therapeutic arsenal. Nevertheless, there will always be patients not responding to these treatments or presenting contraindications, so that alternatives remain necessary.…”

Section: Dear Editormentioning

confidence: 99%

“…Nevertheless, there will always be patients not responding to these treatments or presenting contraindications, so that alternatives remain necessary. One of such valuable alternatives is danazol, which Chang et al 1 Overall, danazol appears as a good choice for the second-line treatment of ITP (especially in older patients) and is clearly at least as efficient as most of the other possibilities. A dosing of 200-400 mg daily is generally well tolerated.…”

Section: Dear Editormentioning

confidence: 99%

Danazol as a second‐line treatment for immune thrombocytopenic purpura

Zimmer

2018

European J of Haematology

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The retrospective study by Chang et al 1 describes the characteristics of a cohort of 375 ITP patients. The first conclusion is that corticosteroids are efficient as a first-line option, which is not really a surprise. The paper then focuses on two second-line treatments, splenectomy, and azathioprine. Whereas the former has long been the reference for chronic/refractory ITP, 2 the latter is used much more rarely and is not even mentioned in a timely review of "old" ITP medications. 3 However, there are some reports about its efficiency in the literature. 4,5 The authors obtain a surprisingly high rate of success and explain the discrepancy with former studies with "difference in case enrollment and definition of response". 1The outcome of a high number of ITP cases has improved since the addition of rituximab and thrombopoietin receptor agonists to the therapeutic arsenal. Nevertheless, there will always be patients not responding to these treatments or presenting contraindications, so that alternatives remain necessary. One of such valuable alternatives is danazol, which Chang et al 1 do not mention, but which was effective in almost all of the studies having included sufficient patient numbers. The series of Ahn et al 6 reports about 96 patients of whom 61.4% achieved a platelet count of ≥50 x 10 9 /L. Among the 26 patients described by Fermand et al, 7 51.4% responded and entered remission. In 2004, Maloisel et al 8 published a study of 57 patients with an overall response rate (defined as a platelet count above 50 x 10 9 /L) of 67% sustained for at least 2 months. More recently, Liu et al 9 saw an overall response rate of 65% in a cohort of 294 patients (103 treated with danazol alone and 191 with danazol and corticosteroids). In a metaanalysis of the effect of immunomodulatory medications for chronic ITP, danazol reached an overall response rate of 58%, the definition of response being in this case a platelet count >30 x 10 9 /L. 10 Finally, Feng et al tested the combination of danazol with all-trans retinoic acid compared to danazol alone in a multicentre, randomized, open-label, phase 2 trial and observed a response rate at 12 months of 62% with the combination therapy vs 28% in the danazol only arm. 11Overall, danazol appears as a good choice for the second-line treatment of ITP (especially in older patients) and is clearly at least as efficient as most of the other possibilities. A dosing of 200-400 mg daily is generally well tolerated. 9,11 However, platelet counts do usually not increase immediately after the introduction of danazol. An additional medication might be initially required. Danazol is less expensive than rituximab.Nevertheless, caution is necessary because of the contraindications (particularly pregnancy but also liver diseases, current or past thromboembolism, hormone-dependent cancers) and potential side effects of this androgen, especially in female patients, like hirsutism, weight gain, or acne. Further adverse reactions include pseudotumor cerebri, stroke, hepatotoxicity, but their i...

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Immune thrombocytopenia: Effectiveness of frontline steroids and comparison of azathioprine, splenectomy, and rituximab as second‐line treatment

Abstract: Steroids are effective frontline treatment for ITP, but relapse is common. Both azathioprine and splenectomy are effective treatment after steroid failure. Rituximab appears to a reasonable second-line treatment option in our limited experience.

Cited by 19 publications

References 46 publications

Reference guide for management of adult immune thrombocytopenia in Japan: 2019 Revision

Reference guide for management of adult immune thrombocytopenia in Japan: 2019 Revision

Treatments for Primary Immune Thrombocytopenia: A Review

Danazol as a second‐line treatment for immune thrombocytopenic purpura

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