Image by Erhabor Osaro
SAN DIEGO—The US Thrombotic Microangiopathies (USTMA) Consortium is planning a longitudinal study to identify biomarkers of relapse and neurocognitive dysfunction in acquired thrombotic thrombocytopenic purpura (TTP).
Earlier studies suggested that ADAMTS13 activity during remission has
limited utility in estimating relapse risk, and cognitive dysfunction
observed in TTP appears to be progressive and independent of relapses.
However, these studies were small with minimal longitudinal data.
Now, the USTMA Consortium, with 13 large referral centers for thrombotic microangiopathies (TMAs) in the US, is providing “the infrastructure for larger US studies in TTP with the goal of improving outcomes in TMAs,” said Marshall A. Mazepa, MD, of the University of Minnesota in Minneapolis.
He described the efforts of the consortium and its plans to conduct a long-term study at the first TTP-TMA Workshop, which took place immediately before the 2016 ASH Annual Meeting.
Dr Mazepa said the objective of the study is to identify biomarkers that
predict the danger period for neurocognitive dysfunction and relapse.
“Hematologists usually do not realize the
neurocognitive dysfunction among patients with TTP, and it is important
that they [hematologists] talk to neurologists,” said Spero Cataland, MD, an organizer of the TTP-TMA Workshop from The Ohio State University in
Columbus.
“This study will be crucial in
improving our understanding of this issue.”
The consortium plans to study 100 TTP patients in remission for 3 years.
Every
3 months, patients will complete neuropsychologic testing, and
investigators will evaluate patients’ end-organ ischemic biomarkers,
ADAMTS13 activity, ultra-large von Willebrand factor levels, and
complement activation biomarkers.
In addition to this study, the USTMA Consortium is participating in the phase 3 HERCULES study of caplacizumab for acquired TTP.
Caplacizumab, which is being developed by Ablynx, was previously evaluated for acquired TTP in the phase 2 TITAN study.
The phase 3 study is a double-blind, placebo-controlled trial designed to determine whether neurocognitive function can be preserved with caplacizumab intervention.
Investigators intend to enroll 92 patients at clinical sites in 17 countries. Recruitment is expected to be complete by the end of 2017.
In closing, Dr Mazepa invited interested clinicians to join the USTMA Consortium by contacting him (mmazepa@umn.edu) or Dr Cataland (spero.cataland@osumc.edu).
