Shared decision making, thrombotic thrombocytopenic purpura, and caplacizumab

Sukumar, Senthil; George, James N.; Cataland, Spero R.

doi:10.1002/ajh.25715

Cited by 27 publications

(35 citation statements)

References 5 publications

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“…Two recent case reports have suggested that even treatment without PEX is feasible in selected patients albeit off label. 8,9 In the German cohort, we report on 6 instances with acute aTTP disease activity treated without PEX. As a consequence, the necessity and duration of plasmapheresis to treat acute episodes of aTTP is a matter of debate.…”

Section: Discussionmentioning

confidence: 99%

Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura

et al. 2020

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Abstract Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare but life-threatening condition. In 2018, the nanobody caplacizumab was approved for the treatment of adults experiencing an acute episode of aTTP, in conjunction with plasma exchange (PEX) and immunosuppression for a minimum of 30 days after stopping daily PEX. We performed a retrospective, observational analysis on the use of caplacizumab in 60 patients from 29 medical centers in Germany during acute disease management. Caplacizumab led to a rapid normalization of the platelet count (median, 3 days; mean 3.78 days). One patient died after late treatment initiation due to aTTP-associated complications. In 2 patients with initial disease presentation and in 4 additional patients with laboratory signs of an exacerbation or relapse after the initial therapy, PEX-free treatment regimens could be established with overall favorable outcome. Caplacizumab is efficacious in the treatment of aTTP independent of timing and ancillary treatment modalities. Based on this real-world experience and published literature, we propose to administer caplacizumab immediately to all patients with an acute episode of aTTP. Treatment decisions regarding the use of PEX should be based on the severity of the clinical presentation and known risk factors. PEX might be dispensable in some patients.

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Section: Discussionmentioning

confidence: 99%

Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura

et al. 2020

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“…Moreover, recent reports have described PEX-free treatment regimens of acute episodes of aTTP with caplacizumab with an overall favorable outcome. 5,6 With this growing body of evidence, caplacizumab has become more and more widely accepted and is recommended as front-line treatment for acute episodes of aTTP.…”

Section: Introductionmentioning

confidence: 99%

ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP

et al. 2020

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Abstract Introduction of the nanobody caplacizumab was shown to be effective in the treatment of acquired thrombotic thrombocytopenic purpura (aTTP) in the acute setting. The official recommendations include plasma exchange (PEX), immunosuppression, and the use of caplacizumab for a minimum of 30 days after stopping daily PEX. This study was a retrospective, observational analysis of the use of caplacizumab in 60 patients from 29 medical centers in Germany. Immunosuppressive treatment led to a rapid normalization of ADAMTS13 activities (calculated median, 21 days). In 35 of 60 patients, ADAMTS13 activities started to normalize before day 30 after PEX; in 11 of 60 patients, the treatment was extended beyond day 30; and in 5 patients, it was extended even beyond day 58 due to persistent autoimmune activity. In 34 of 60 instances, caplacizumab was stopped before day 30 with a favorable outcome whenever ADAMTS13 activities were >10%. In contrast, 11 of 34 patients with ADAMTS13 activities <10% at the time of stopping caplacizumab treatment developed a nonfavorable outcome (disease exacerbation or relapse). In some cases, prolongation of the treatment interval to every other day was feasible and resulted in a sustained reduction of von Willebrand factor activity. ADAMTS13 activity measurements are central for a rapid diagnosis in the acute setting but also to tailor disease management. An ADAMTS13 activity–guided approach seems safe for identifying the individual time point when to stop caplacizumab to prevent overtreatment and undertreatment; this approach will result in significant cost savings without jeopardizing the well-being of patients. In addition, von Willebrand factor activity may serve as a biomarker for drug monitoring.

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“…Therefore, this collection of patients is heterogeneous in many aspects. All patients received caplacizumab immediately once aTTP was suspected or diagnosed, and plasma exchange was omitted based on shared decision making between patient and the treating physicians because of poor venous access (n = 2), oligosymptomatic presentation (n = 4), and refusal of consent to plasma exchange (n = 1) 8 . Patients and/or relatives were informed about the characteristics of their disease and the advantages and disadvantages of possible and available treatment options including central venous line placement and plasma exchange therapy.…”

Section: Resultsmentioning

confidence: 99%

“…[4][5][6] Recent reports have shown that plasma exchange-free treatment is feasible, but have limitations because ADAMTS13 activities were not reported at the time when caplacizumab was first administered, or the aTTP episode was rather mild. 7,8…”

Section: Introductionmentioning

confidence: 99%

“…However, with the advent of this potent novel drug and in view of potentially life‐threatening procedure‐associated complications, the role of plasma exchange in the treatment of aTTP remains to be reevaluated 4‐6 . Recent reports have shown that plasma exchange‐free treatment is feasible, but have limitations because ADAMTS13 activities were not reported at the time when caplacizumab was first administered, or the aTTP episode was rather mild 7,8 …”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Treatment of acquired thrombotic thrombocytopenic purpura without plasma exchange in selected patients under caplacizumab

Völker

Brinkkoetter

Knöbl

et al. 2020

Journal of Thrombosis and Haemostasis

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Shared decision making, thrombotic thrombocytopenic purpura, and caplacizumab

Cited by 27 publications

References 5 publications

Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura

Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura

ADAMTS13 and VWF activities guide individualized caplacizumab treatment in patients with aTTP

Treatment of acquired thrombotic thrombocytopenic purpura without plasma exchange in selected patients under caplacizumab

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