Multiagent induction and maintenance therapy for patients with refractory immune thrombocytopenic purpura (ITP)

Boruchov, Donna; Gururangan, S.; Driscoll, Monica; Bussel, James B.

doi:10.1182/blood-2007-01-065763

Cited by 88 publications

(74 citation statements)

References 29 publications

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“…Another study describes the success of combinations of IVIg, steroids, vincristine, and anti-D for remission induction (25/35 [71%]) and combination azathioprine and danazol for maintenance (13/17 [76.5%]); however, only half of the patients in that study had failed splenectomy. 7 Our study provides one approach for the severely refractory ITP patient who has limited treatment options. The use of lower doses of cyclosporine helps reduce the need for frequent monitoring and parenteral administration can be avoided for all drugs; 2 issues that help simplify treatment.…”

Section: Resultsmentioning

confidence: 99%

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Combination immunosuppressant therapy for patients with chronic refractory immune thrombocytopenic purpura

Arnold

Nazi

Santos

et al. 2010

Blood

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Treatment options for patients with chronic refractory immune thrombocytopenic purpura (ITP) are limited. Because combination immunosuppressant therapy appeared to be effective in ITP and other disorders, we used this approach in patients with particularly severe and refractory ITP. In this retrospective, observational study, we determined the response (platelet count above 30 ؋ 10 9 /L and doubling of baseline) among 19 refractory ITP patients. Treatment consisted of azathioprine, mycophenolate mofetil, and cyclosporine. The patients had failed a median of 6 prior treatments, including splenectomy (in all except 1 IntroductionImmune thrombocytopenic purpura (ITP) is an acquired bleeding disorder characterized by autoantibody-mediated platelet destruction and impaired platelet production. Patients with chronic refractory ITP have the highest risk of death and disease-related or therapy-related complications. 1,2 Treatment options include aggressive immunosuppressant therapy, and most recently thrombopoietin (TPO) receptor agonists. 3,4 Single-agent immunosuppressant drugs such as azathioprine and cyclosporine have been used to treat refractory patients with moderate success 5 ; however dose escalation can cause morbidity, and other options are needed.Over the past several decades, physicians have noted that greater efficacy can be achieved using a combination of unrelated but synergistic medications. 6,7 In this report, we describe our experience using a combination of azathioprine, mycophenolate mofetil, and cyclosporine to treat patients with particularly severe and refractory ITP. MethodsPatients in this report had a platelet count less than 20 ϫ 10 9 /L that persisted for at least 12 months with an inadequate or transient response to multiple therapies. The senior author (J.G.K.) offered the option of a combination of immunosuppressant therapy. Patients with comorbidities such as liver failure or uncontrolled hypertension were not offered this treatment. Institutional Review Board approval from McMaster University was obtained to retrospectively review the medical charts of all patients with ITP treated in our clinic; this report describes only those patients treated with combination immunosuppressant therapy. Institutional Review Board approval was not required for the administration of the combination of immunosuppressant agents (each on its own an accepted therapy for ITP 8 ), which was given per clinical need.Medical records of each patient were reviewed by 3 independent assessors and data were abstracted in triplicate and verified for consistency. Platelet count measurements and follow-up visits were done as per routine care and mean monthly platelet counts were calculated. Target doses of immunosuppressant medications were azathioprine 2 mg/kg per day; mycophenolate mofetil 1 to 2 g/d; and cyclosporine 2 mg/kg per day. Low-dose cyclosporine was chosen to minimize toxicity and avoid the need for drug level monitoring.We defined overall response as a platelet count level of 30 ϫ 10 9 /L or higher and do...

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Section: Resultsmentioning

confidence: 99%

“…6,7 In this report, we describe our experience using a combination of azathioprine, mycophenolate mofetil, and cyclosporine to treat patients with particularly severe and refractory ITP.…”

Section: Introductionmentioning

confidence: 99%

Combination immunosuppressant therapy for patients with chronic refractory immune thrombocytopenic purpura

Arnold

Nazi

Santos

et al. 2010

Blood

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“…15 TPO-A increases the platelet count via stimulating megakaryocytopoiesis and thereby increasing thrombopoiesis to a level that overcomes the antiplatelet antibody effect in the majority of chronic ITP patients. [21][22][23] The pathoetiology of nonresponse to TPO-A in patients with ITP has not been well studied, and the mechanisms are currently unknown. Theoretically, they could range from defects at the level of the TPO receptor, including its signaling pathway, to increased thrombopoiesis insufficient to overcome peripheral platelet destruction.…”

Section: Introductionmentioning

confidence: 99%

Platelet production and platelet destruction: assessing mechanisms of treatment effect in immune thrombocytopenia

Barsam

Psaila

Forestier

et al. 2011

Blood

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145

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“…Azathioprine [60] 2 mg/kg PO daily 30-90 d 30-180 d AMG53 [61,41,42] 3-10 ug/kg weekly SC 5-14 d 14-60 d…”

Section: -14 D 4-28 Dmentioning

confidence: 99%

A Case Report of Bernard-Soulier Syndrome in Differential Diagnosis of Immune Thrombocytopenic Purpura.

Wasfi¹,

ijar²

2018

IJAR

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Multiagent induction and maintenance therapy for patients with refractory immune thrombocytopenic purpura (ITP)

Cited by 88 publications

References 29 publications

Combination immunosuppressant therapy for patients with chronic refractory immune thrombocytopenic purpura

Combination immunosuppressant therapy for patients with chronic refractory immune thrombocytopenic purpura

Platelet production and platelet destruction: assessing mechanisms of treatment effect in immune thrombocytopenia

A Case Report of Bernard-Soulier Syndrome in Differential Diagnosis of Immune Thrombocytopenic Purpura.

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