Autoantibody-resistant ADAMTS13 variant

Miyata, Toshiyuki

doi:10.1182/blood.2021011147

Cited by 1 publication

(3 citation statements)

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“…For iTTP, the strategy of overriding autoantibodies with the wild‐type rhADAMTS13 123–125 allows for speculations on a ‘bio‐better’ rhADAMTS13 that would be resistant against a large fraction of most patients' autoantibody mixtures (Figure 3). 49,126,128 We recently reported a full‐length ADAMTS13 variant with an artificially introduced N ‐glycan bearing some of the in vitro characteristics of this idealized rhADAMTS13 ‘bio‐better’ 127,129 . Although further studies are still required before drawing more definite conclusions, the current scientific evidence is encouraging. Raising the bar in the elderly is a current goal, since age does widen the risk for premature death of iTTP patients 108 .…”

Section: The Targets On the Road – Perspectives About The Future In Ttpmentioning

confidence: 92%

“…49 , 126 , 128 We recently reported a full‐length ADAMTS13 variant with an artificially introduced N ‐glycan bearing some of the in vitro characteristics of this idealized rhADAMTS13 ‘bio‐better’. 127 , 129 Although further studies are still required before drawing more definite conclusions, the current scientific evidence is encouraging. Raising the bar in the elderly is a current goal, since age does widen the risk for premature death of iTTP patients.…”

Section: The Targets On the Road – Perspectives About The Future In ...mentioning

confidence: 98%

“…127 Despite the current lack of in vivo data, rhADAMTS13 ‘bio‐betters’ like these could possibly be used with some anticipated advantages for the reduction of the burden of care (Figure 3 ). 128 , 129 …”

Section: Milestones In Ttp Treatment: Meet the New...mentioning

confidence: 99%

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TTP: From empiricism for an enigmatic disease to targeted molecular therapies

et al. 2022

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The 100th anniversary of the first description of Thrombotic Thrombocytopenic Purpura (TTP) as a disease by Dr. Eli Moschcowitz approaches. For many decades, TTP remained mostly a mysterious fatal condition, where diagnosis was often postmortem. Initially a pentad of symptoms was identified, a pattern that later revealed to be fallible. Sporadic observations led to empiric interventions that allowed for the first impactful breakthrough in TTP treatment, almost 70 years after its first description: the introduction of plasma exchange and infusions as treatments. The main body of knowledge within the field was gathered in the latest three decades: patient registries were set and proved crucial for advancements; the general mechanisms of disease have been described; the diagnosis was refined; new treatments and biomarkers with improvements on prognosis and management were introduced.Further changes and improvements are expected in the upcoming decades. In this review, we provide a brief historic overview of TTP, as an illustrative example of the success of translational medicine enabling to rapidly shift from a management largely based on empiricism to targeted therapies and personalized medicine, for the benefit of patients. Current management options and present and future perspectives in this still evolving field are summarized.

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