HEALTHCARE RESOURCE UTILIZATION WITH RECOMBINANT ADAMTS13 IN PATIENTS WITH CONGENITAL TTP: RESULTS FROM A PHASE 3 RANDOMIZED CLINICAL TRIAL
Author(s)
Marie Scully, MD1, Paul Knöbl, MD2, Paul Coppo, MD, PhD3, Wolf Achim Hassenpflug, MD4, Satoshi Higasa, MD5, Santiago Bonanad, MD6, Sami Ibrahimi, MD7, Shan Xiao, PhD8, Tara M. Robinson, MD, PhD8, Dorothy Romanus, PhD8, Jerzy Windyga, PhD, MD9;
1Department of Haematology, University College London Hospital, Haematology Theme-NIHR UCLH/UCL BRC, London, United Kingdom, 2Division of Hematology and Hemostasis, Medical University of Vienna, Vienna, Austria, 3Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne – Université (AP-HP.6), Paris, France, 4Department of Pediatric Hematology and Oncology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany, 5Department of Hematology, Hyogo College of Medicine Hospital, Nishinomiya, Japan, 6Hemostasis and Thrombosis Unit, Department of Hematology, Hospital Universitari i Politècnic La Fe, Valencia, Spain, 7Department of Medicine, Section of Hematology and Medical Oncology, University of Oklahoma Health Sciences Center - Stephenson Cancer Center, Oklahoma City, OK, USA, 8Takeda Development Center Americas, Inc., Cambridge, MA, USA, 9Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
1Department of Haematology, University College London Hospital, Haematology Theme-NIHR UCLH/UCL BRC, London, United Kingdom, 2Division of Hematology and Hemostasis, Medical University of Vienna, Vienna, Austria, 3Department of Hematology and National Reference Center for Thrombotic Microangiopathies, Hôpital Saint-Antoine, Assistance Publique-Hôpitaux de Paris and Sorbonne – Université (AP-HP.6), Paris, France, 4Department of Pediatric Hematology and Oncology, University Medical Centre Hamburg-Eppendorf, Hamburg, Germany, 5Department of Hematology, Hyogo College of Medicine Hospital, Nishinomiya, Japan, 6Hemostasis and Thrombosis Unit, Department of Hematology, Hospital Universitari i Politècnic La Fe, Valencia, Spain, 7Department of Medicine, Section of Hematology and Medical Oncology, University of Oklahoma Health Sciences Center - Stephenson Cancer Center, Oklahoma City, OK, USA, 8Takeda Development Center Americas, Inc., Cambridge, MA, USA, 9Department of Hemostasis Disorders and Internal Medicine, Laboratory of Hemostasis and Metabolic Diseases, Institute of Hematology and Transfusion Medicine, Warsaw, Poland
OBJECTIVES: A prospective, randomized, controlled, open-label phase 3 trial (NCT03393975) investigated the efficacy and safety of recombinant ADAMTS13 (rADAMTS13; Takeda Pharmaceuticals U.S.A., Inc.) and plasma-based therapy (PBT) prophylaxis in congenital thrombotic thrombocytopenic purpura (cTTP). Here we present healthcare resource utilization (HCRU) data from this trial.
METHODS: Patients 0-70 years old with confirmed cTTP were randomized 1:1 to receive either 40 IU/kg rADAMTS13 every 1-2 weeks or PBT prophylaxis, as dictated by the participant’s usual PBT prophylaxis schedule. Participants received treatment for 6 months (Period 1) and then crossed over to the alternate treatment for 6 months (Period 2); all participants then received rADAMTS13 prophylaxis for 6 months (Period 3). HCRU assessments included the number and duration of hospital admissions during prophylaxis overall and for acute TTP events. All participants provided written informed consent.
RESULTS: Forty-seven participants received a mean ±SD number of 35.0 ±12.36 infusions of rADAMTS13 (Periods 1-3) and 48 received 16.9 ±6.48 infusions of PBT (Periods 1-2); mean infusion duration 5.49 ±3.61 and 144.94 ±85.15 minutes, respectively. The mean ±SD annualized number of hospital admissions for any reason was 0.25 ±0.70 for rADAMTS13 (n=44) and 2.79 ±6.48 for PBT (n=45) during Periods 1-2, and 0.42 ±0.87 for rADAMTS13 (n=44) throughout Periods 1-3. The mean ±SD annualized hospital duration was 0.84 ±2.32 days/year for rADAMTS13 and 8.10 ±20.32 days/year for PBT (Periods 1-2), and 2.99 ±11.04 days/year for rADAMTS13 throughout Periods 1-3. The mean ±SD annualized number of hospital admissions for acute TTP events was 0.00 ±0.00 for rADAMTS13 and 0.04 ±0.26 for PBT (Periods 1-2), and 0.00 ±0.00 for rADAMTS13 throughout Periods 1-3.
CONCLUSIONS: Patients receiving rADAMTS13 experienced a shorter infusion duration and lower annualized hospitalization rates and duration, indicating lower HCRU and treatment burden for rADAMTS13 compared with those receiving PBT.
METHODS: Patients 0-70 years old with confirmed cTTP were randomized 1:1 to receive either 40 IU/kg rADAMTS13 every 1-2 weeks or PBT prophylaxis, as dictated by the participant’s usual PBT prophylaxis schedule. Participants received treatment for 6 months (Period 1) and then crossed over to the alternate treatment for 6 months (Period 2); all participants then received rADAMTS13 prophylaxis for 6 months (Period 3). HCRU assessments included the number and duration of hospital admissions during prophylaxis overall and for acute TTP events. All participants provided written informed consent.
RESULTS: Forty-seven participants received a mean ±SD number of 35.0 ±12.36 infusions of rADAMTS13 (Periods 1-3) and 48 received 16.9 ±6.48 infusions of PBT (Periods 1-2); mean infusion duration 5.49 ±3.61 and 144.94 ±85.15 minutes, respectively. The mean ±SD annualized number of hospital admissions for any reason was 0.25 ±0.70 for rADAMTS13 (n=44) and 2.79 ±6.48 for PBT (n=45) during Periods 1-2, and 0.42 ±0.87 for rADAMTS13 (n=44) throughout Periods 1-3. The mean ±SD annualized hospital duration was 0.84 ±2.32 days/year for rADAMTS13 and 8.10 ±20.32 days/year for PBT (Periods 1-2), and 2.99 ±11.04 days/year for rADAMTS13 throughout Periods 1-3. The mean ±SD annualized number of hospital admissions for acute TTP events was 0.00 ±0.00 for rADAMTS13 and 0.04 ±0.26 for PBT (Periods 1-2), and 0.00 ±0.00 for rADAMTS13 throughout Periods 1-3.
CONCLUSIONS: Patients receiving rADAMTS13 experienced a shorter infusion duration and lower annualized hospitalization rates and duration, indicating lower HCRU and treatment burden for rADAMTS13 compared with those receiving PBT.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HSD118
Topic
Health Service Delivery & Process of Care
Disease
SDC: Rare & Orphan Diseases