ArticleAdvances in therapy2026
Estimating Long-Term Organ Damage Risk in Patients with Congenital Thrombotic Thrombocytopenic Purpura Receiving Recombinant ADAMTS13 Prophylaxis: A Structured Expert Elicitation Study.
Article in Advances in therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionLongitudinal 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.
methodsSix 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.
resultsIn 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.
conclusionExperts 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.
Indexed as
Identifiers
42853532What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.