Efficacy of mycophenolate mofetil as single‐agent therapy for refractory immune thrombocytopenic purpura

Provan, Drew; Moss, Ashley J.; Newland, Adrian C.; Bussel, James B.

doi:10.1002/ajh.20515

Cited by 80 publications

(52 citation statements)

References 36 publications

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“…Our findings contrast with several reports or clinical studies showing prolonged response with such therapies, but patients showed less refractory disease than our patients. [13][14][15][16][17][18][19] Despite the efficacy of HSCT, the high risk of mortality associated with this procedure (1 death in our cohort) argues for its proposal for patients with no alternative therapeutic option only. However, the combination of Tpo-RAs with immunosuppressant drugs was effective with an overall response rate (R 1 CR) of 70% in the 10 patients who received this treatment.…”

Section: Discussionmentioning

confidence: 83%

Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia

Mahévas

Gerfaud‐Valentin

Moulis

et al. 2016

Blood

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Key Points• The baseline characteristics of multirefractory ITP differed from "typical" ITP, outcome was severe, and was associated with high morbidity and mortality.• Combining immunosuppressant therapy with a thrombopoietinreceptor agonist may be a relevant option for these patients.Refractory immune thrombocytopenia (ITP) was previously defined as lack of a minimum response to splenectomy and the requirement for long-term treatment to reduce the risk of significant bleeding events. In this multicenter study, we included 37 patients with multirefractory ITP, defined as no response to splenectomy, rituximab, romiplostim, and eltrombopag. As compared with a historical cohort of 183 ITP patients, matched on the calendar year of ITP diagnosis with a 5:1 ratio, patients with multirefractory ITP were more likely to have secondary ITP (odds ratio [OR], 4.84; 95% confidence interval [CI], 1.31-17.86; P 5 .018) and monoclonal gammopathy of undetermined significance (OR, 5.94; 95% CI, 1.08-32.48; P 5 .04). The median duration of ITP before being recognized as multirefractory was 78 months (range, 6-450). The patients showed failure of a median of 10.5 prior treatment lines for ITP (range, 6-15). At the end of follow-up (median, 84 months; range, 12-455), only 1/14 patients achieved response with immunosuppressant therapy alone. By contrast, 7/10 patients achieved response with a combination of immunosuppressant therapy and thrombopoietin-receptor agonists that lasted for a median of 15 months (range, 6-32). Throughout the course of ITP, 5/37 patients died, 3 with ITP (bleeding, n 5 2; sepsis n 5 1); 15 (40%) had at least 1 bacterial infection and 9 (24%) at least 1 episode of thrombosis. In conclusion, multirefractory ITP was associated with high morbidity and mortality. Combining an immunosuppressant therapy with thrombopoietin-receptor agonists may be a good strategy for management for these patients with severe disease. (Blood. 2016;128(12):1625-1630

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Section: Discussionmentioning

confidence: 83%

Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia

Mahévas

Gerfaud‐Valentin

Moulis

et al. 2016

Blood

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“…[76][77][78][79][80][81][82] We begin MMF at a dose of 500 mg orally twice per day and increase the dose to 1000 to 1500 mg twice per day after 2 weeks. Protocols that use this approach have demonstrated overall response rates of 50% to 60%.…”

Section: Tiermentioning

confidence: 99%

“…Protocols that use this approach have demonstrated overall response rates of 50% to 60%. [76][77][78][79][80][81] Durability of response after discontinuation of therapy is variable. 77,79,82 MMF is generally well tolerated; principal adverse effects include headache and gastrointestinal symptoms.…”

Section: Tiermentioning

confidence: 99%

How I treat refractory immune thrombocytopenia

Cuker

Neunert

2016

Blood

129

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This article summarizes our approach to the management of children and adults with primary immune thrombocytopenia (ITP) who do not respond to, cannot tolerate, or are unwilling to undergo splenectomy. We begin with a critical reassessment of the diagnosis and a deliberate attempt to exclude nonautoimmune causes of thrombocytopenia and secondary ITP. For patients in whom the diagnosis is affirmed, we consider observation without treatment.Observation is appropriate for most asymptomatic patients with a platelet count of 20 to 30 3 10 9 /L or higher. We use a tiered approach to treat patients who require therapy to increase the platelet count. Tier 1 options (rituximab, thrombopoietin receptor agonists, low-dose corticosteroids) have a relatively favorable therapeutic index. We exhaust all Tier 1 options before proceeding to Tier 2, which comprises a host of immunosuppressive agents with relatively lower response rates and/or greater toxicity. We often prescribe Tier 2 drugs not alone but in combination with a Tier 1 or a second Tier 2 drug with a different mechanism of action. We reserve Tier 3 strategies, which are of uncertain benefit and/or high toxicity with little supporting evidence, for the rare patient with serious bleeding who does not respond to Tier 1 and Tier 2 therapies. (Blood. 2016;128(12): 1547-1554 Case 1A 10-year-old male was diagnosed with primary immune thrombocytopenia (ITP) 5 months ago when he presented with epistaxis, petechiae, bruising, and a platelet count of 5 3 10 9 /L. He responded transiently to intravenous immunoglobulin G (IgG), but epistaxis recurred 2 weeks later. He subsequently received a short course of oral corticosteroids to which he had a temporary response in platelet count and cessation of epistaxis. Since discontinuing corticosteroids, he has had only occasional bruising and petechiae. His platelet count is currently 13 3 10 9 /L. He states that ITP is not interfering with activities.

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“…About 40% of splenectomized patients either do not respond or relapse after surgery [6], approximately 10% of patients have early perioperative or delayed infections, and some develop surgical complications, although rarely fatal. In case of relapse or resistant to corticosteroids ITP, other immunosuppressive drugs can be used including azathioprine, cyclosporine A, cyclophosphamide, danazol, mycophenolate mofetil, all with marginal efficacy [7][8][9][10][11]. Intravenous immunoglobulin (IVIG) and anti-D are also used in the treatment of ITP, especially when rapid improvement of platelet count is needed (in case of life-threatening bleeding or prior to splenectomy or other surgical procedures).…”

Section: Open    Accessmentioning

confidence: 99%

Maintenance Therapy with Rituximab in Adult Patients with Immune Thrombocytopenia

et al. 2013

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Immune thrombocytopenia (ITP) is an autoimmune disease of unknown etiology, characterized by isolated thrombocytopenia and the absence of any underlying cause for thrombocytopenia. Corticosteroids are the standard first line treatment for patients with symptomatic disease, inducing platelet count recovery in 70-80% of patients; but in many cases, steroid tapering or withdrawal is followed by a decrease of platelet count and the need for additional treatment. Splenectomy is still the standard salvage therapy in cases refractory to corticosteroid therapy. In the past decade monoclonal anti-CD20 antibodies (Rituximab) are being increasingly used in patients with refractory ITP and other autoimmune diseases. Recent studies show that Rituximab is useful in the treatment of patients with chronic and refractory ITP. We report two cases with chronic ITP treated with standard dose of Rituximab in four weekly doses and than continue to receive maintenance therapy with Rituximab for 2 years.

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Efficacy of mycophenolate mofetil as single‐agent therapy for refractory immune thrombocytopenic purpura

Cited by 80 publications

References 36 publications

Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia

Characteristics, outcome, and response to therapy of multirefractory chronic immune thrombocytopenia

How I treat refractory immune thrombocytopenia

Maintenance Therapy with Rituximab in Adult Patients with Immune Thrombocytopenia

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