Daratumumab for refractory immune‐mediated thrombotic thrombocytopenic purpura

Aggarwal, Asha; White, Danielle; Pavord, Sue; Thomas, Will; Desborough, Michael

doi:10.1111/bjh.18854

Cited by 6 publications

(1 citation statement)

References 16 publications

(27 reference statements)

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“…Over the last few years, there have been quite a few case reports and case series of the successful use of bortezomib in patients with refractory iTTP and it has come to be regarded as a safe and efective treatment option in this patient population [11][12][13][14][15]. Bortezomib, a proteasome inhibitor, is FDA-approved for use in multiple myeloma as well as mantle cell lymphoma.…”

Section: Discussionmentioning

confidence: 99%

Successful Use of Bortezomib in an Adolescent with Refractory TTP

Wali,

Quigley,

Schaefer

2023

Case Reports in Hematology

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With increasing early and upfront use of rituximab and caplacizumab in the modern management of immune-mediated thrombotic thrombocytopenic purpura (iTTP), the risk of refractory disease is expected to decline. However, despite the use of adequate initial therapy, a small subset of patients develop a refractory disease which is difficult to manage. Bortezomib has come to be known as a safe and effective treatment option for refractory iTTP, but its use in children is limited. Here, we describe the case of an adolescent patient with refractory iTTP who had a satisfactory and sustained response to the use of bortezomib.

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Section: Discussionmentioning

confidence: 99%

Successful Use of Bortezomib in an Adolescent with Refractory TTP

Wali,

Quigley,

Schaefer

2023

Case Reports in Hematology

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Multiple drugs

2023

Reactions Weekly

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Bortezomib in relapsed/refractory immune thrombotic thrombocytopenic purpura: A single‐centre retrospective cohort and systematic literature review

et al. 2023

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SummaryImmune thrombotic thrombocytopenic purpura (iTTP) is a rare and life‐threatening haematological condition. Initial treatment involves plasma exchange (PLEX), corticosteroids, caplacizumab and rituximab. In relapsed and refractory cases despite initial treatments, further immune‐modulating therapy includes the proteasome inhibitor, bortezomib. Evidence for bortezomib in this setting is limited to case reports and case series. We report our experience and perform a systematic review of the literature. We identified 21 publications with 28 unique patients in addition to our cohort of eight patients treated with bortezomib. The median age of patients was 44 years (IQR: 27–53) and 69% female. They were usually in an initial, refractory presentation of iTTP where they had received PLEX, corticosteroids, rituximab and another line of therapy. After bortezomib administration, 72% of patients had a complete response, with 85% maintaining a durable response without relapse at the last follow‐up.

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Daratumumab for refractory immune‐mediated thrombotic thrombocytopenic purpura

Cited by 6 publications

References 16 publications

Successful Use of Bortezomib in an Adolescent with Refractory TTP

Successful Use of Bortezomib in an Adolescent with Refractory TTP

Multiple drugs

Bortezomib in relapsed/refractory immune thrombotic thrombocytopenic purpura: A single‐centre retrospective cohort and systematic literature review

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