Advances in therapy · 2026
Estimating Long-Term Organ Damage Risk in Patients with Congenital Thrombotic Thrombocytopenic Purpura Receiving Recombinant ADAMTS13 Prophylaxis: A Structured Expert Elicitation Study.
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Padhiar Amie, Burke Colin, Puls Mathilde, Berkley Annie, Heard Oliver, Kaur Harneet, Wolff Harold, Murphy Linda
Аннотация
INTRODUCTION: Longitudinal real-world data in congenital thrombotic thrombocytopenic purpura (cTTP) are scarce. Structured expert elicitation (SEE) was conducted to estimate the projected risk of long-term organ damage in children and adolescents and in adult patients with cTTP receiving recombinant ADAMTS13 (rADAMTS13) prophylaxis, without history of organ damage.
METHODS: Six UK clinicians with experience in managing cTTP provided individual judgments via an online SEE survey. The probability of developing ≥ 1 long-term organ damage outcomes, including major organ events such as stroke/transient ischemic attack, myocardial infarction, and chronic kidney disease, at predefined ages was estimated. Responses were collected using the quartile method and combined via linear opinion pooling.
RESULTS: In children and adolescents, median (95% credible interval) estimated cumulative long-term organ damage projected risks were 3.25% (1.09-20.26%) by age 15 years, 4.48% (0.86-16.11%) by 20 years, and 5.76% (0.96-19.20%) by 25 years. In adults, estimated risks were 4.85% (0.59-16.96%) by 45 years and 7.42% (1.47-29.29%) by 55 years.
CONCLUSION: Experts consistently projected lower long-term risks of organ damage with continuous rADAMTS13 prophylaxis, although these estimates remain hypothesis-generating. These expert-derived estimates provide structured insights into potential long-term outcomes associated with rADAMTS13 prophylaxis, which can inform future research, health technology assessments, and evidence-generation efforts in cTTP. Prospective empirical studies are required to confirm the findings. Graphical Abstract available for this article.
METHODS: Six UK clinicians with experience in managing cTTP provided individual judgments via an online SEE survey. The probability of developing ≥ 1 long-term organ damage outcomes, including major organ events such as stroke/transient ischemic attack, myocardial infarction, and chronic kidney disease, at predefined ages was estimated. Responses were collected using the quartile method and combined via linear opinion pooling.
RESULTS: In children and adolescents, median (95% credible interval) estimated cumulative long-term organ damage projected risks were 3.25% (1.09-20.26%) by age 15 years, 4.48% (0.86-16.11%) by 20 years, and 5.76% (0.96-19.20%) by 25 years. In adults, estimated risks were 4.85% (0.59-16.96%) by 45 years and 7.42% (1.47-29.29%) by 55 years.
CONCLUSION: Experts consistently projected lower long-term risks of organ damage with continuous rADAMTS13 prophylaxis, although these estimates remain hypothesis-generating. These expert-derived estimates provide structured insights into potential long-term outcomes associated with rADAMTS13 prophylaxis, which can inform future research, health technology assessments, and evidence-generation efforts in cTTP. Prospective empirical studies are required to confirm the findings. Graphical Abstract available for this article.