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Mycophenolate Mofetil for first-line treatment of Immune Thrombocytopenia
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Bradbury, Charlotte A., Pell, Julie, Hill, Quentin, Bagot, Catherine, Cooper, Nichola, Ingram, Jenny, Breheny, Katie, Kandiyali, Rebecca, Rayment, Rachel, Evans, Gillian, Talks, Kate, Thomas, Ian and Greenwood, Rosemary (2021) Mycophenolate Mofetil for first-line treatment of Immune Thrombocytopenia. New England Journal Of Medicine , 385 (10). pp. 885-895. doi:10.1056/NEJMoa2100596 ISSN 0028-4793.
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Official URL: http://dx.doi.org/10.1056/NEJMoa2100596
Abstract
BACKGROUND: Immune thrombocytopenia (ITP) is a rare autoimmune thrombocytopenia with associated bleeding risk and fatigue. Recommended first line ITP treatment is with high dose glucocorticoids but side effects, heterogeneous responses and high relapse rates are significant problems.
METHODS: In this multicenter, UK based, open label, randomized controlled trial, we randomly assigned adult patients with ITP requiring first line treatment to glucocorticoid alone (standard care) versus combined glucocorticoid and mycophenolate (1:1 ratio). The primary efficacy outcome was time from randomization to treatment failure, defined as platelets <30x109/L with initiation of 2nd line treatment. Secondary outcomes included response rates, side effects, bleeding events, patient reported outcome measures and serious adverse events. RESULTS: One hundred and twenty ITP patients were recruited and randomized (52.4% male, mean age 54 years, range 17-87, mean platelets = 7x109/L), and followed up for up to 2 years. Fewer treatment failures were seen in patients randomized to mycophenolate (22%, n=13/59 vs 44%, n=27/61, aHR=0.41 [0.21, 0.80], p=0.0064), with higher response rates (91.5% achieving platelets>100x109/L vs 63.9%, p<0.001). We found no evidence of difference between groups in bleeding events, rescue treatments or treatment side effects including infection. However, quality of life measures of physical function and fatigue were worse in the mycophenolate group. CONCLUSIONS Addition of mycophenolate to glucocorticoid for first line ITP treatment results in higher response rates and reduces the risk of refractory or relapsed ITP but with somewhat decreased quality of life.
Item Type: | Journal Article | ||||||||||||
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Divisions: | Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School > Clinical Trials Unit Faculty of Science, Engineering and Medicine > Medicine > Warwick Medical School |
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Journal or Publication Title: | New England Journal Of Medicine | ||||||||||||
Publisher: | Massachusetts Medical Society | ||||||||||||
ISSN: | 0028-4793 | ||||||||||||
Official Date: | 2 September 2021 | ||||||||||||
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Volume: | 385 | ||||||||||||
Number: | 10 | ||||||||||||
Page Range: | pp. 885-895 | ||||||||||||
DOI: | 10.1056/NEJMoa2100596 | ||||||||||||
Status: | Peer Reviewed | ||||||||||||
Publication Status: | Published | ||||||||||||
Access rights to Published version: | Restricted or Subscription Access | ||||||||||||
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