Therapeutic switch from plasma to recombinant ADAMTS13 for patients with congenital TTP from Japanese real-world data

Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultrarare disorder characterized by thrombocytopenia, microangiopathic hemolytic anemia, and ischemic organ damage caused by pathogenic ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif, member 13) variants. ADAMTS13-containing product, including fresh-frozen plasma (FFP), and plasma-derived factor VIII concentrates are commonly used to supply ADAMTS13; however, frequent hospital visits and allergic reactions are major drawbacks. A recombinant ADAMTS13 (rADAMTS13) was recently developed to address these issues. However, real-world evidence has not been reported owing to the rarity of this condition. This study compared the efficacy and safety of FFP and rADAMTS13 in 14 Japanese patients, including 5 patients with end-stage renal disease who were excluded from the phase 3 trial. The median peak level of ADAMTS13 activity 15 minutes after rADAMTS13 administration was significantly higher than that after FFP (68.4% vs 15.9%; P < .001). ADAMTS13 activity 1 week after rADAMTS13 administration was well maintained compared with FFP infusion (11.6% vs 5.1%; P < .001). Patients reported no allergic reactions after rADAMTS13 administration and appreciated the convenience of a single infusion of rADAMTS13, suggesting that rADAMTS13 is a safe and effective alternative to FFP in patients with cTTP. To our knowledge, this is the first publication of patients with cTTP who switched FFP to novel rADAMTS13 from Japanese real-world data.

Authors

Institutions

Publication Details

Journal
Blood
Published
2025-03-16
DOI
https://doi.org/10.1182/blood.2024027516
Citations
9
Primary Topic
Complement system in diseases
Type
article
Field-Weighted Citation Impact
7.18
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Therapeutic switch from plasma to recombinant ADAMTS13 for patients with congenital TTP from Japanese real-world data

Hidekazu Azumi, Kazuya Sakai, Kenki Saito, Yoshiko Yoshimura et al.
9 citations
Blood
Complement system in diseases
7.18
article

Therapeutic switch from plasma to recombinant ADAMTS13 for patients with congenital TTP from Japanese real-world data

Hidekazu Azumi, Kazuya Sakai, Kenki Saito, Yoshiko Yoshimura, Miyuki Abe, Atsushi Hamamura, Naoki Fujita, Fumiyoshi Ikejiri, Katsuyoshi Koh, Yoshiyuki Ogawa, Tazuko Tokugawa, Masanori Matsumoto, Norimichi Hattori, Masahiro Takeyama, Minoru Kanaya, Hiroshi Shiragami, Kazuki Tanaka, Rie Kanai, Teruhisa Taoka
article en
9 citations

Abstract

Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultrarare disorder characterized by thrombocytopenia, microangiopathic hemolytic anemia, and ischemic organ damage caused by pathogenic ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif, member 13) variants. ADAMTS13-containing product, including fresh-frozen plasma (FFP), and plasma-derived factor VIII concentrates are commonly used to supply ADAMTS13; however, frequent hospital visits and allergic reactions are major drawbacks. A recombinant ADAMTS13 (rADAMTS13) was recently developed to address these issues. However, real-world evidence has not been reported owing to the rarity of this condition. This study compared the efficacy and safety of FFP and rADAMTS13 in 14 Japanese patients, including 5 patients with end-stage renal disease who were excluded from the phase 3 trial. The median peak level of ADAMTS13 activity 15 minutes after rADAMTS13 administration was significantly higher than that after FFP (68.4% vs 15.9%; P < .001). ADAMTS13 activity 1 week after rADAMTS13 administration was well maintained compared with FFP infusion (11.6% vs 5.1%; P < .001). Patients reported no allergic reactions after rADAMTS13 administration and appreciated the convenience of a single infusion of rADAMTS13, suggesting that rADAMTS13 is a safe and effective alternative to FFP in patients with cTTP. To our knowledge, this is the first publication of patients with cTTP who switched FFP to novel rADAMTS13 from Japanese real-world data.

BloodVol. 145(20)
SHOWA Medical University (JP), Hyogo Medical University (JP), Gunma University (JP), Osaka National Hospital (JP), Otsu Municipal Hospital (JP), Saitama Children's Medical Center (JP), Kisarazu Hospital (JP), Saiseikai Shimonoseki General Hospital (JP), Sakai Municipal Hospital (JP), Higashiosaka College & Higashiosaka Junior College (JP), Children's Medical Center (IR), Sapporo Hokuyu Hospital (JP), Nara Medical University Hospital (JP), Higashi Osaka City General Hospital (JP), Shimane Prefectural Central Hospital (JP), Izumi City General Hospital (JP), Usui (Japan) (JP), Beppu Medical Center (JP), National Hospital Organization (JP), Izumo Science Center (JP), Hyogo Prefectural Nishinomiya Hospital (JP), Nara Medical University (JP)
Good health and well-being
Openalex Percentile: Top 3%
Complement system in diseases
7.18
DOISourceOpenAlex
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.