Strong association between insufficient plasma exchange and fatal outcomes in Japanese patients with immune-mediated thrombotic thrombocytopenic purpura

Kayashima, Michinori; Sakai, Kazuya; Harada, Kazuki; Kanetake, Jun; Kubo, Masayuki; Hamada, Eriko; Hayakawa, Masaki; Hatakeyama, Kinta; Matsumoto, Masanori

doi:10.1007/s12185-021-03197-5

Cited by 9 publications

(3 citation statements)

References 29 publications

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“…As for treatments for immune-mediated TTP, prompt and adequate plasma exchange [119][120][121] and corticosteroid therapy have been used [122]. Plasma exchange can remove unusually large VWF multimers, remove anti-ADAMTS13 antibodies, and replenish fresh ADAMTS13 [123].…”

Section: Ttpmentioning

confidence: 99%

How We Interpret Thrombosis with Thrombocytopenia Syndrome?

Yamada,

Asakura

2024

IJMS

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Platelets play an important role in hemostasis, and a low platelet count usually increases the risk of bleeding. Conditions in which thrombosis occurs despite low platelet counts are referred to as thrombosis with thrombocytopenia syndrome, including heparin-induced thrombocytopenia, vaccine-induced immune thrombotic thrombocytopenia, paroxysmal nocturnal hemoglobinuria, antiphospholipid syndrome, thrombotic microangiopathy (TMA), and disseminated intravascular coagulation. TMA includes thrombotic thrombocytopenic purpura, Shiga toxin-producing Escherichia coli-associated hemolytic uremic syndrome (HUS), and atypical HUS. Patients with these pathologies present with thrombosis and consumptive thrombocytopenia associated with the activation of platelets and the coagulation system. Treatment varies from disease to disease, and many diseases have direct impacts on mortality and organ prognosis if therapeutic interventions are not promptly implemented. Underlying diseases and the results of physical examinations and general laboratory tests as part of a thorough workup for patients should promptly lead to therapeutic intervention before definitive diagnosis. For some diseases, the diagnosis and initial treatment must proceed in parallel. Utilization of not only laboratory tests but also various scoring systems is important for validating therapeutic interventions based on clinical information.

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Section: Ttpmentioning

confidence: 99%

How We Interpret Thrombosis with Thrombocytopenia Syndrome?

Yamada,

Asakura

2024

IJMS

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“…A Japanese retrospective study revealed that 26 out of 32 patients experienced sudden death, mostly following radical hypotension and bradycardia. The median follow-up time after admission was 5.0 days, and nine patients underwent autopsy and had cardiac microvascular thrombi in arterioles [57]. The UK group reported a retrospective study on cardiac involvement in acute iTTP.…”

Section: Troponin-t/i and Glasgow Coma Score And Their Link With Acut...mentioning

confidence: 99%

ADAMTS13 and Non-ADAMTS13 Biomarkers in Immune-Mediated Thrombotic Thrombocytopenic Purpura

Bonnez,

Sakai,

Vanhoorelbeke

2023

JCM

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Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare medical emergency for which a correct and early diagnosis is essential. As a severe deficiency in A Disintegrin And Metalloproteinase with ThromboSpondin type 1 repeats, member 13 (ADAMTS13) is the underlying pathophysiology, diagnostic strategies require timely monitoring of ADAMTS13 parameters to differentiate TTP from alternative thrombotic microangiopathies (TMAs) and to guide initial patient management. Assays for conventional ADAMTS13 testing focus on the enzyme activity and presence of (inhibitory) anti-ADAMTS13 antibodies to discriminate immune-mediated TTP (iTTP) from congenital TTP and guide patient management. However, diagnosis of iTTP remains challenging when patients present borderline ADAMTS13 activity. Therefore, additional biomarkers would be helpful to support correct clinical judgment. Over the last few years, the evaluation of ADAMTS13 conformation has proven to be a valuable tool to confirm the diagnosis of acute iTTP when ADAMST13 activity is between 10 and 20%. Screening of ADAMTS13 conformation during long-term patient follow-up suggests it is a surrogate marker for undetectable antibodies. Moreover, some non-ADAMTS13 parameters gained notable interest in predicting disease outcome, proposing meticulous follow-up of iTTP patients. This review summarizes non-ADAMTS13 biomarkers for which inclusion in routine clinical testing could largely benefit differential diagnosis and follow-up of iTTP patients.

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“…The risk of relapse is estimated to be about 40-55% over the 7-8 years following an acute event [2,3], or about 2% per month [4]. In a US registry study, the 20-year mortality rate was approximately 20% among individuals with iTTP [2], and in Japan, the 12-month rate of iTTP-related mortality has been reported as approximately 13% [5].…”

Section: Introductionmentioning

confidence: 99%

The efficacy and safety of caplacizumab in Japanese patients with immune-mediated thrombotic thrombocytopenic purpura: an open-label phase 2/3 study

et al. 2022

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Caplacizumab is an anti-von Willebrand factor humanized single-variable-domain immunoglobulin fragment whose efficacy and safety in immune-mediated thrombotic thrombocytopenia purpura (iTTP) have been demonstrated in international studies. This prospective, open-label phase 2/3 study evaluated caplacizumab 10 mg administered daily during plasma exchange and for 30 days afterward, in combination with immunosuppressive treatment, in Japanese adults with a clinical diagnosis of iTTP (new or recurrent). The primary endpoint was prevention of iTTP recurrence; key secondary endpoints included time to platelet count response, time to organ damage normalization, and safety. Among 21 treated patients, 1 of 15 (6.7%) evaluable patients developed iTTP recurrence. Median time to normalization was 2.79 days for platelet count and 2.65 days for organ damage markers (n = 15). Treatment-emergent adverse events (TEAEs) were mostly mild to moderate in severity; the most frequently reported caplacizumab-related TEAEs were increased alanine aminotransferase, epistaxis, and gastrointestinal hemorrhage (all in 9.5% of patients). At least one bleeding event was reported in 7 of 21 patients (33%). Caplacizumab was effective in Japanese patients with iTTP, with a low rate of iTTP recurrence, rapid normalization of platelet counts and organ damage markers, and no unexpected TEAEs. Trial registration: ClinicalTrials.gov identifier, NCT04074187.

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Strong association between insufficient plasma exchange and fatal outcomes in Japanese patients with immune-mediated thrombotic thrombocytopenic purpura

Cited by 9 publications

References 29 publications

How We Interpret Thrombosis with Thrombocytopenia Syndrome?

How We Interpret Thrombosis with Thrombocytopenia Syndrome?

ADAMTS13 and Non-ADAMTS13 Biomarkers in Immune-Mediated Thrombotic Thrombocytopenic Purpura

The efficacy and safety of caplacizumab in Japanese patients with immune-mediated thrombotic thrombocytopenic purpura: an open-label phase 2/3 study

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