Bortezomib in relapsed/refractory immune thrombotic thrombocytopenic purpura: A single-centre retrospective cohort and systematic literature review.
Br J Haematol
; 204(2): 638-643, 2024 02.
Article
in En
| MEDLINE
| ID: mdl-37571963
ABSTRACT
Immune 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.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Purpura, Thrombotic Thrombocytopenic
/
Purpura, Thrombocytopenic, Idiopathic
Type of study:
Systematic_reviews
Limits:
Adult
/
Female
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Humans
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Male
/
Middle aged
Language:
En
Journal:
Br J Haematol
Year:
2024
Document type:
Article
Affiliation country: