Caplacizumab for PE-free TTP Treatment - MAYARI - Prof. Paul Coppo, Prof. Paul Brinkkötter
youtu.be/oaofdqJ1EN4?...
youtu.be/oaofdqJ1EN4?...
Caplacizumab for PE-free TTP Treatment - MAYARI - Prof. Paul Coppo, Prof. Paul Brinkkötter
YouTube video by MARKUS at HOMe
youtu.be
February 8, 2026 at 7:28 PM
Caplacizumab for PE-free TTP Treatment - MAYARI - Prof. Paul Coppo, Prof. Paul Brinkkötter
youtu.be/oaofdqJ1EN4?...
youtu.be/oaofdqJ1EN4?...
Excellent piece illustrating contradictions of our medical #innovation ecosystem, socializing risks/costs while privatizing profits, through example of innovative nanobody drug from VUB/VIB in Belgium, and offering an alternative through #publicpharma healthpolicy-watch.news/medicine-for...
Medicine For Rare Disorder Provides Case Study Of Contradictions In Drug Development System - Health Policy Watch
The journey of the medicine, Caplacizumab – from a publicly funded scientific breakthrough to a high-cost pharmaceutical product controlled by a multinational
healthpolicy-watch.news
April 1, 2025 at 1:12 PM
Excellent piece illustrating contradictions of our medical #innovation ecosystem, socializing risks/costs while privatizing profits, through example of innovative nanobody drug from VUB/VIB in Belgium, and offering an alternative through #publicpharma healthpolicy-watch.news/medicine-for...
Medicine for Rare Disorder Provides Case Study of Contradictions in Drug Development System
Inside View | David Franco
@viblifesciences.bsky.social
healthpolicy-watch.news/medicine-for...
Inside View | David Franco
@viblifesciences.bsky.social
healthpolicy-watch.news/medicine-for...
Medicine For Rare Disorder Provides Case Study Of Contradictions In Drug Development System - Health Policy Watch
The journey of the medicine, Caplacizumab – from a publicly funded scientific breakthrough to a high-cost pharmaceutical product controlled by a multinational
healthpolicy-watch.news
April 7, 2025 at 3:14 AM
Medicine for Rare Disorder Provides Case Study of Contradictions in Drug Development System
Inside View | David Franco
@viblifesciences.bsky.social
healthpolicy-watch.news/medicine-for...
Inside View | David Franco
@viblifesciences.bsky.social
healthpolicy-watch.news/medicine-for...
Studie: Provrörsexperiment med antikroppsliknande molekyler. Man började med en s k nanokropp (små antikroppar i kameldjur & hajar), ympade den på en godkänd nanokroppsplattform (Caplacizumab) för att få ner immunogeniciteten, och satte ihop dem 3 och 3.🤷♂️
www.biorxiv.org/content/10.1...
www.biorxiv.org/content/10.1...
Significantly enhancing antiviral efficacy by computational optimization and structural engineering of grafted nanobodies
Since the outbreak of the COVID-19 pandemic in 2019, antibody-based therapies have played a key role in combating the virus. In addition to conventional antibodies, single-domain heavy-chain antibodie...
www.biorxiv.org
November 19, 2024 at 2:26 PM
Studie: Provrörsexperiment med antikroppsliknande molekyler. Man började med en s k nanokropp (små antikroppar i kameldjur & hajar), ympade den på en godkänd nanokroppsplattform (Caplacizumab) för att få ner immunogeniciteten, och satte ihop dem 3 och 3.🤷♂️
www.biorxiv.org/content/10.1...
www.biorxiv.org/content/10.1...
💉 Prvo zdravilo na osnovi nanoteles 🧬
Leta 2018 je caplacizumab postal prvo zdravilo za zdravljenje trombotične trombocitopenične purpure. Z njegovim odobravanjem sta Ameriška in Evropska agencija za zdravila naredili pomemben preboj za uporabo nanoteles v medicini. 365.rtvslo.si/arhiv/ugrizn...
Leta 2018 je caplacizumab postal prvo zdravilo za zdravljenje trombotične trombocitopenične purpure. Z njegovim odobravanjem sta Ameriška in Evropska agencija za zdravila naredili pomemben preboj za uporabo nanoteles v medicini. 365.rtvslo.si/arhiv/ugrizn...
Nanotelesa – novo orožje medicine: Prvo nanotelesno zdravilo
Prvo nanotelesno zdravilo, caplacizumab, je bilo namenjeno zdravljenju trombotične trombocitopenične purpure (TTP). Ta smrtonosna bolezen povzroča nastajanje krvnih strdkov v najmanjših žilah in lahko...
365.rtvslo.si
February 14, 2025 at 10:46 AM
💉 Prvo zdravilo na osnovi nanoteles 🧬
Leta 2018 je caplacizumab postal prvo zdravilo za zdravljenje trombotične trombocitopenične purpure. Z njegovim odobravanjem sta Ameriška in Evropska agencija za zdravila naredili pomemben preboj za uporabo nanoteles v medicini. 365.rtvslo.si/arhiv/ugrizn...
Leta 2018 je caplacizumab postal prvo zdravilo za zdravljenje trombotične trombocitopenične purpure. Z njegovim odobravanjem sta Ameriška in Evropska agencija za zdravila naredili pomemben preboj za uporabo nanoteles v medicini. 365.rtvslo.si/arhiv/ugrizn...
🔬 [NEWS]
Une étude menée par le consortium international sur le PTT et autres MAT, à laquelle la France a grandement participé, montre que le caplacizumab, lorsqu'il est administré tôt, améliore la survie et réduit les complications.
🗞 pubmed.ncbi.nlm.nih.gov/40235949/
#PTT #MaladiesRares #FSMR
Une étude menée par le consortium international sur le PTT et autres MAT, à laquelle la France a grandement participé, montre que le caplacizumab, lorsqu'il est administré tôt, améliore la survie et réduit les complications.
🗞 pubmed.ncbi.nlm.nih.gov/40235949/
#PTT #MaladiesRares #FSMR
April 23, 2025 at 8:00 AM
🔬 [NEWS]
Une étude menée par le consortium international sur le PTT et autres MAT, à laquelle la France a grandement participé, montre que le caplacizumab, lorsqu'il est administré tôt, améliore la survie et réduit les complications.
🗞 pubmed.ncbi.nlm.nih.gov/40235949/
#PTT #MaladiesRares #FSMR
Une étude menée par le consortium international sur le PTT et autres MAT, à laquelle la France a grandement participé, montre que le caplacizumab, lorsqu'il est administré tôt, améliore la survie et réduit les complications.
🗞 pubmed.ncbi.nlm.nih.gov/40235949/
#PTT #MaladiesRares #FSMR
Early intervention with plasma exchange, steroids, and rituximab boosts iTTP survival to 93%, but caplacizumab raises bleeding risk.
by Pishko AM, Li A and Cuker A in JAMA #MedSky
👉 get more here
📖 read the article: https://jamanetwork.com/journals/jama/article-abstract/2834159
by Pishko AM, Li A and Cuker A in JAMA #MedSky
👉 get more here
📖 read the article: https://jamanetwork.com/journals/jama/article-abstract/2834159
August 21, 2025 at 6:39 PM
Early intervention with plasma exchange, steroids, and rituximab boosts iTTP survival to 93%, but caplacizumab raises bleeding risk.
by Pishko AM, Li A and Cuker A in JAMA #MedSky
👉 get more here
📖 read the article: https://jamanetwork.com/journals/jama/article-abstract/2834159
by Pishko AM, Li A and Cuker A in JAMA #MedSky
👉 get more here
📖 read the article: https://jamanetwork.com/journals/jama/article-abstract/2834159
📰 #iTTP is a rare disease that primarily affects individuals aged 20-40 years. Authors show results indicate caplacizumab is effective in reducing refractoriness, risk of death & likelihood of exacerbations in elderly patients, similar to outcomes observed in younger patients 👉https://bit.ly/42h2a4H
July 16, 2025 at 11:01 AM
📰 #iTTP is a rare disease that primarily affects individuals aged 20-40 years. Authors show results indicate caplacizumab is effective in reducing refractoriness, risk of death & likelihood of exacerbations in elderly patients, similar to outcomes observed in younger patients 👉https://bit.ly/42h2a4H
The HemosIL AcuStar CLIA rapidly and accurately identifies TTP, effectively preventing unnecessary treatments for patients without the condition.
by Deshpande SR, Tarawneh H (...) Cuker A et 4 al. in Blood #MedSky
👉 get more here
📖 read the article:
by Deshpande SR, Tarawneh H (...) Cuker A et 4 al. in Blood #MedSky
👉 get more here
📖 read the article:
Rapid ADAMTS13 activity assays for thrombotic thrombocytopenic purpura: a systematic review and meta-analysis
Key Points. Rapid ADAMTS13 assays have turnaround times within one hour and potential to avert empiric TPE and caplacizumab in patients without TTPThe Hemo
ashpublications.org
April 30, 2025 at 12:55 PM
ADAMTS13 70% 就不是 TTP
確診 TTP 需要活性 <10%。血漿置換 7 天後測到 70%，代表當初的診斷方向要重新檢討 —— 應停止血漿置換，回頭找其他 TMA 原因（aHUS、次發性 TMA、惡性腫瘤相關）。
繼續置換並加 caplacizumab 或 rituximab 是錯誤的方向。
#血液專科
https://hema-2026.hsiehting.com/q/110-078
確診 TTP 需要活性 <10%。血漿置換 7 天後測到 70%，代表當初的診斷方向要重新檢討 —— 應停止血漿置換，回頭找其他 TMA 原因（aHUS、次發性 TMA、惡性腫瘤相關）。
繼續置換並加 caplacizumab 或 rituximab 是錯誤的方向。
#血液專科
https://hema-2026.hsiehting.com/q/110-078
August 4, 2026 at 7:38 AM
ADAMTS13 70% 就不是 TTP
確診 TTP 需要活性 <10%。血漿置換 7 天後測到 70%，代表當初的診斷方向要重新檢討 —— 應停止血漿置換，回頭找其他 TMA 原因（aHUS、次發性 TMA、惡性腫瘤相關）。
繼續置換並加 caplacizumab 或 rituximab 是錯誤的方向。
#血液專科
https://hema-2026.hsiehting.com/q/110-078
確診 TTP 需要活性 <10%。血漿置換 7 天後測到 70%，代表當初的診斷方向要重新檢討 —— 應停止血漿置換，回頭找其他 TMA 原因（aHUS、次發性 TMA、惡性腫瘤相關）。
繼續置換並加 caplacizumab 或 rituximab 是錯誤的方向。
#血液專科
https://hema-2026.hsiehting.com/q/110-078
Caplacizumab 不是取代 ADAMTS13
它是 anti-VWF 的 nanobody，阻斷 VWF A1 domain 與血小板 GPIb 的交互作用，把「血小板黏上去」這一步擋掉。
它不分解 ULVWF，也不影響抗體生成，所以必須併用血漿置換與免疫抑制，不能單獨使用。
#血液專科
https://hema-2026.hsiehting.com/q/113-017
它是 anti-VWF 的 nanobody，阻斷 VWF A1 domain 與血小板 GPIb 的交互作用，把「血小板黏上去」這一步擋掉。
它不分解 ULVWF，也不影響抗體生成，所以必須併用血漿置換與免疫抑制，不能單獨使用。
#血液專科
https://hema-2026.hsiehting.com/q/113-017
August 3, 2026 at 12:02 PM
Caplacizumab 不是取代 ADAMTS13
它是 anti-VWF 的 nanobody，阻斷 VWF A1 domain 與血小板 GPIb 的交互作用，把「血小板黏上去」這一步擋掉。
它不分解 ULVWF，也不影響抗體生成，所以必須併用血漿置換與免疫抑制，不能單獨使用。
#血液專科
https://hema-2026.hsiehting.com/q/113-017
它是 anti-VWF 的 nanobody，阻斷 VWF A1 domain 與血小板 GPIb 的交互作用，把「血小板黏上去」這一步擋掉。
它不分解 ULVWF，也不影響抗體生成，所以必須併用血漿置換與免疫抑制，不能單獨使用。
#血液專科
https://hema-2026.hsiehting.com/q/113-017
三種 TMA 的分流
ADAMTS13 <10% → TTP，治療是血漿置換加免疫抑制（可加 caplacizumab）。
前驅血性腹瀉、補體正常 → STEC-HUS，支持性治療為主，不建議抗補體。
兩者都不是 → aHUS，治療用 eculizumab 或 ravulizumab。
#血液專科
https://hema-2026.hsiehting.com/q/114-065
ADAMTS13 <10% → TTP，治療是血漿置換加免疫抑制（可加 caplacizumab）。
前驅血性腹瀉、補體正常 → STEC-HUS，支持性治療為主，不建議抗補體。
兩者都不是 → aHUS，治療用 eculizumab 或 ravulizumab。
#血液專科
https://hema-2026.hsiehting.com/q/114-065
August 3, 2026 at 9:40 AM
三種 TMA 的分流
ADAMTS13 <10% → TTP，治療是血漿置換加免疫抑制（可加 caplacizumab）。
前驅血性腹瀉、補體正常 → STEC-HUS，支持性治療為主，不建議抗補體。
兩者都不是 → aHUS，治療用 eculizumab 或 ravulizumab。
#血液專科
https://hema-2026.hsiehting.com/q/114-065
ADAMTS13 <10% → TTP，治療是血漿置換加免疫抑制（可加 caplacizumab）。
前驅血性腹瀉、補體正常 → STEC-HUS，支持性治療為主，不建議抗補體。
兩者都不是 → aHUS，治療用 eculizumab 或 ravulizumab。
#血液專科
https://hema-2026.hsiehting.com/q/114-065
Caplacizumab 的角色與代價
Nanobody，阻斷血小板與 VWF 結合，可加速血小板正常化、降低 30 天內早期復發（風險差 −29%），但出血風險上升（風險差 +17%）。
它不影響抗 ADAMTS13 抗體的產生，所以必須併用類固醇與 rituximab，否則停藥後晚期復發。
#血液專科
https://hema-2026.hsiehting.com/q/114-027
Nanobody，阻斷血小板與 VWF 結合，可加速血小板正常化、降低 30 天內早期復發（風險差 −29%），但出血風險上升（風險差 +17%）。
它不影響抗 ADAMTS13 抗體的產生，所以必須併用類固醇與 rituximab，否則停藥後晚期復發。
#血液專科
https://hema-2026.hsiehting.com/q/114-027
August 3, 2026 at 8:25 AM
Caplacizumab 的角色與代價
Nanobody，阻斷血小板與 VWF 結合，可加速血小板正常化、降低 30 天內早期復發（風險差 −29%），但出血風險上升（風險差 +17%）。
它不影響抗 ADAMTS13 抗體的產生，所以必須併用類固醇與 rituximab，否則停藥後晚期復發。
#血液專科
https://hema-2026.hsiehting.com/q/114-027
Nanobody，阻斷血小板與 VWF 結合，可加速血小板正常化、降低 30 天內早期復發（風險差 −29%），但出血風險上升（風險差 +17%）。
它不影響抗 ADAMTS13 抗體的產生，所以必須併用類固醇與 rituximab，否則停藥後晚期復發。
#血液專科
https://hema-2026.hsiehting.com/q/114-027
Studie: Provrörsexperiment med antikroppsliknande molekyler. Man började med en s k nanokropp (små antikroppar i kameldjur & hajar), ympade den på en godkänd nanokroppsplattform (Caplacizumab) för att få ner immunogeniciteten, och satte ihop dem 3 och 3.🤷♂️ […]
Original post on mastodon.social
mastodon.social
November 19, 2024 at 7:16 PM
Studie: Provrörsexperiment med antikroppsliknande molekyler. Man började med en s k nanokropp (små antikroppar i kameldjur & hajar), ympade den på en godkänd nanokroppsplattform (Caplacizumab) för att få ner immunogeniciteten, och satte ihop dem 3 och 3.🤷♂️ […]
Might kick this off with a short and sweet piece about TTP and pregnancy.
TTP is always a time for swift action but it doesn’t have to be scary:
(1) Stop clots fast
(2) Multidisciplinary decision making
(3) Best thing for bub is a healthy mum
TTP is always a time for swift action but it doesn’t have to be scary:
(1) Stop clots fast
(2) Multidisciplinary decision making
(3) Best thing for bub is a healthy mum
TTP and pregnancy
Following the diagnosis of thrombocytopenic purpura (TTP) in pregnancy, plasma exchange and steroids should be initiated. Rituximab can be used and caplacizumab can be considered. In subsequent pregn...
onlinelibrary.wiley.com
November 10, 2024 at 9:17 PM
Might kick this off with a short and sweet piece about TTP and pregnancy.
TTP is always a time for swift action but it doesn’t have to be scary:
(1) Stop clots fast
(2) Multidisciplinary decision making
(3) Best thing for bub is a healthy mum
TTP is always a time for swift action but it doesn’t have to be scary:
(1) Stop clots fast
(2) Multidisciplinary decision making
(3) Best thing for bub is a healthy mum
Open Access UCL Research: Caplacizumab resistance in immune thrombotic thrombocytopenic purpura (iTTP) associated with VWF A1 domain missense variant discovery.ucl.ac.uk/id/eprint/10...
Caplacizumab resistance in immune thrombotic thrombocytopenic purpura (iTTP) associated with VWF A1 domain missense variant
-
UCL Discovery
UCL Discovery is UCL's open access repository, showcasing and providing access to UCL research outputs from all UCL disciplines.
discovery.ucl.ac.uk
March 11, 2026 at 4:10 PM
Open Access UCL Research: Caplacizumab resistance in immune thrombotic thrombocytopenic purpura (iTTP) associated with VWF A1 domain missense variant discovery.ucl.ac.uk/id/eprint/10...
Caplacizumab Treatment in Elderly Patients with iTTP: Updated Experience from the Spanish TTP Registry ashpublications.org/blood/articl... #hvhebron #hemat
Caplacizumab Treatment in Elderly Patients with iTTP: Updated Experience from the Spanish TTP Registry
Disclosures. De La Rubia:Janssen: Honoraria, Speakers Bureau; GSK: Honoraria, Research Funding, Speakers Bureau; Pfizer: Speakers Bureau; Sanofi: Speakers
ashpublications.org
April 17, 2025 at 2:52 PM
Caplacizumab Treatment in Elderly Patients with iTTP: Updated Experience from the Spanish TTP Registry ashpublications.org/blood/articl... #hvhebron #hemat
Feed: "Health Policy Watch"
By: Kerry Cullinan on Monday, March 31, 2025
By: Kerry Cullinan on Monday, March 31, 2025
Medicine for Rare Disorder Provides Case Study of Contradictions in Drug Development System
The journey of the medicine, Caplacizumab – from a publicly funded scientific breakthrough to a high-cost pharmaceutical product controlled by a multinational
healthpolicy-watch.news
April 1, 2025 at 6:20 AM
Feed: "Health Policy Watch"
By: Kerry Cullinan on Monday, March 31, 2025
By: Kerry Cullinan on Monday, March 31, 2025
Feed: "CheckRare"
By: Madaline Spencer on Thursday, February 27, 2025
By: Madaline Spencer on Thursday, February 27, 2025
Caplacizumab Combination Therapy in Patients With Acquired Thrombotic Thrombocytopenic Purpura
Alix Arnaud, Sanofi, discusses the effects of caplacizumab combination therapy in patients with acquired TTP.
checkrare.com
February 27, 2025 at 1:16 PM
Feed: "CheckRare"
By: Madaline Spencer on Thursday, February 27, 2025
By: Madaline Spencer on Thursday, February 27, 2025
This is how you journal club. #caplacizumab #ttp
December 5, 2024 at 12:47 AM
This is how you journal club. #caplacizumab #ttp
According to findings from a small retrospective study assessing refractoriness in #iTTP treated with #caplacizumab, just 1.5% of patients met the laboratory criteria for true refractoriness, and this was only noted to occur in the context of confounding factors: https://ow.ly/3jfH50YR7b2
#HemeSky
#HemeSky
April 30, 2026 at 1:01 PM
According to findings from a small retrospective study assessing refractoriness in #iTTP treated with #caplacizumab, just 1.5% of patients met the laboratory criteria for true refractoriness, and this was only noted to occur in the context of confounding factors: https://ow.ly/3jfH50YR7b2
#HemeSky
#HemeSky
#VisualAbstract TTP, once nearly always fatal, now has a survival rate over 95% due to advances in understanding ADAMTS13 deficiency and treatments like plasma exchange, immunosuppression, caplacizumab, and recombinant ADAMTS13. zurl.co/wZ3uw
August 8, 2025 at 4:00 PM
#VisualAbstract TTP, once nearly always fatal, now has a survival rate over 95% due to advances in understanding ADAMTS13 deficiency and treatments like plasma exchange, immunosuppression, caplacizumab, and recombinant ADAMTS13. zurl.co/wZ3uw
TTP, once nearly always fatal, now has a survival rate over 95% due to advances in understanding ADAMTS13 deficiency and treatments like plasma exchange, immunosuppression, caplacizumab, and recombinant ADAMTS13. zurl.co/jwQnP
July 28, 2025 at 8:00 AM
TTP, once nearly always fatal, now has a survival rate over 95% due to advances in understanding ADAMTS13 deficiency and treatments like plasma exchange, immunosuppression, caplacizumab, and recombinant ADAMTS13. zurl.co/jwQnP