Bortezomib in relapsed/refractory immune thrombotic thrombocytopenic purpura : A single-centre retrospective cohort and systematic literature review
© 2023 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd..
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.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2024 |
|---|---|
| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:204 |
|---|---|
| Enthalten in: |
British journal of haematology - 204(2024), 2 vom: 01. Feb., Seite 638-643 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Lee, Nicholas C J [VerfasserIn] |
|---|
| Links: |
|---|
| Anmerkungen: |
Date Completed 08.02.2024 Date Revised 05.03.2024 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1111/bjh.19035 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM360714595 |
|---|
| LEADER | 01000caa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM360714595 | ||
| 003 | DE-627 | ||
| 005 | 20240306232350.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2024 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1111/bjh.19035 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1318.xml |
| 035 | |a (DE-627)NLM360714595 | ||
| 035 | |a (NLM)37571963 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Lee, Nicholas C J |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Bortezomib in relapsed/refractory immune thrombotic thrombocytopenic purpura |b A single-centre retrospective cohort and systematic literature review |
| 264 | 1 | |c 2024 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 08.02.2024 | ||
| 500 | |a Date Revised 05.03.2024 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2023 The Authors. British Journal of Haematology published by British Society for Haematology and John Wiley & Sons Ltd. | ||
| 520 | |a 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 | ||
| 650 | 4 | |a Systematic Review | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a ADAMTS13 | |
| 650 | 4 | |a TTP | |
| 650 | 4 | |a bortezomib | |
| 650 | 4 | |a immune thrombotic thrombocytopenic purpura | |
| 650 | 4 | |a systematic review | |
| 650 | 7 | |a Bortezomib |2 NLM | |
| 650 | 7 | |a 69G8BD63PP |2 NLM | |
| 650 | 7 | |a Rituximab |2 NLM | |
| 650 | 7 | |a 4F4X42SYQ6 |2 NLM | |
| 650 | 7 | |a Adrenal Cortex Hormones |2 NLM | |
| 650 | 7 | |a ADAMTS13 Protein |2 NLM | |
| 650 | 7 | |a EC 3.4.24.87 |2 NLM | |
| 700 | 1 | |a Yates, Sean |e verfasserin |4 aut | |
| 700 | 1 | |a Rambally, Siayareh |e verfasserin |4 aut | |
| 700 | 1 | |a Sarode, Ravi |e verfasserin |4 aut | |
| 700 | 1 | |a Ibrahim, Ibrahim F |e verfasserin |4 aut | |
| 700 | 1 | |a Shen, Yu-Min |e verfasserin |4 aut | |
| 700 | 1 | |a Hofmann, Sandra L |e verfasserin |4 aut | |
| 700 | 1 | |a Bavli, Natalie R |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t British journal of haematology |d 1955 |g 204(2024), 2 vom: 01. Feb., Seite 638-643 |w (DE-627)NLM000000396 |x 1365-2141 |7 nnns |
| 773 | 1 | 8 | |g volume:204 |g year:2024 |g number:2 |g day:01 |g month:02 |g pages:638-643 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1111/bjh.19035 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 204 |j 2024 |e 2 |b 01 |c 02 |h 638-643 | ||