PMID- 36397934
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20230328
IS  - 2475-0379 (Electronic)
IS  - 2475-0379 (Linking)
VI  - 6
IP  - 8
DP  - 2022 Nov
TI  - Safety and efficacy of long-term emicizumab prophylaxis in hemophilia A with 
      factor VIII inhibitors: A phase 3b, multicenter, single-arm study (STASEY).
PG  - e12837
LID - 10.1002/rth2.12837 [doi]
LID - e12837
AB  - BACKGROUND: The bispecific monoclonal antibody emicizumab bridges activated 
      factor IX and factor X, mimicking the cofactor function of activated factor VIII 
      (FVIII), restoring hemostasis. OBJECTIVES: The Phase 3b STASEY study was designed 
      to assess the safety of emicizumab prophylaxis in people with hemophilia A (HA) 
      with FVIII inhibitors. METHODS: People with HA received 3 mg/kg emicizumab once 
      weekly (QW) for 4 weeks followed by 1.5 mg/kg QW for 2 years. The primary 
      objective was the safety of emicizumab prophylaxis, including incidence and 
      severity of adverse events (AEs) and AEs of special interest (thrombotic events 
      [TEs] and thrombotic microangiopathies). Secondary objectives included efficacy 
      (annualized bleed rates [ABRs]). RESULTS: Overall, 195 participants were 
      enrolled; 193 received emicizumab. The median (range) duration of exposure was 
      103.1 (1.1-108.3) weeks. Seven (3.6%) participants discontinued emicizumab. The 
      most common AEs were arthralgia (n = 33, 17.1%) and nasopharyngitis (n = 30, 
      15.5%). The most common treatment-related AE was injection-site reaction (n = 19, 
      9.8%). Two fatalities were reported (polytrauma with fatal head injuries and 
      abdominal compartment syndrome); both were deemed unrelated to emicizumab by 
      study investigators. Two TEs occurred (myocardial infarction and localized clot 
      following tooth extraction), also deemed unrelated to emicizumab. The negative 
      binomial regression model-based ABR (95% confidence interval) for treated bleeds 
      was 0.5 (0.27-0.89). Overall, 161 participants (82.6%) had zero treated bleeds. 
      CONCLUSIONS: The safety profile of emicizumab prophylaxis was confirmed in a 
      large population of people with HA with FVIII inhibitors and no new safety 
      signals occurred. The majority of participants had zero treated bleeds.
CI  - (c) 2022 The Authors. Research and Practice in Thrombosis and Haemostasis published 
      by Wiley Periodicals LLC on behalf of International Society on Thrombosis and 
      Haemostasis (ISTH).
FAU - Jimenez-Yuste, Victor
AU  - Jimenez-Yuste V
AUID- ORCID: 0000-0003-3937-3499
AD  - Hospital Universitario La Paz Autonoma University Madrid Spain.
FAU - Peyvandi, Flora
AU  - Peyvandi F
AUID- ORCID: 0000-0001-7423-9864
AD  - IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi 
      Hemophilia and Thrombosis Center Milan Italy.
AD  - Department of Pathophysiology and Transplantation University of Milan Milan 
      Italy.
FAU - Klamroth, Robert
AU  - Klamroth R
AUID- ORCID: 0000-0003-4194-8183
AD  - Comprehensive Care Haemophilia Treatment Centre, Vivantes Klinikum Berlin 
      Germany.
FAU - Castaman, Giancarlo
AU  - Castaman G
AUID- ORCID: 0000-0003-4973-1317
AD  - Careggi University Hospital Florence Italy.
FAU - Shanmukhaiah, Chandrakala
AU  - Shanmukhaiah C
AD  - Department of Haematology Seth GS Medical College and King Edward Memorial 
      Hospital Mumbai India.
FAU - Rangarajan, Savita
AU  - Rangarajan S
AD  - Clinical Trials & Research Unit K J Somaiya Superspeciality Hospital & Research 
      Centre Mumbai India.
AD  - Faculty of Medicine University of Southampton Southampton UK.
FAU - Garcia Chavez, Jaime
AU  - Garcia Chavez J
AD  - Unidad de Investigacion en Enfermedades Hematologicas, Hospital de Especialidades 
      CMN La Raza, IMSS Mexico City Mexico.
FAU - Martinez, Raul
AU  - Martinez R
AD  - UMAE Hospital De Especialidades CMNSXXI Mexico City Mexico.
FAU - Kenet, Gili
AU  - Kenet G
AUID- ORCID: 0000-0003-3494-9823
AD  - National Hemophilia Center, Sheba Medical Center Tel Hashomer Israel.
AD  - The Amalia Biron Research Institute of Thrombosis & Hemostasis Sackler Medical 
      School Tel Aviv University Tel Aviv Israel.
FAU - Alzahrani, Hazaa
AU  - Alzahrani H
AD  - King Faisal Specialist Hospital and Research Centre Riyadh Saudi Arabia.
FAU - Robson, Susan
AU  - Robson S
AD  - F. Hoffmann-La Roche Ltd Basel Switzerland.
FAU - Schmitt, Christophe
AU  - Schmitt C
AD  - F. Hoffmann-La Roche Ltd Basel Switzerland.
FAU - Kiialainen, Anna
AU  - Kiialainen A
AD  - F. Hoffmann-La Roche Ltd Basel Switzerland.
FAU - Meier, Oliver
AU  - Meier O
AD  - F. Hoffmann-La Roche Ltd Basel Switzerland.
FAU - Ozelo, Margareth
AU  - Ozelo M
AD  - Hemocentro UNICAMP University of Campinas Campinas Brazil.
LA  - eng
PT  - Journal Article
DEP - 20221114
PL  - United States
TA  - Res Pract Thromb Haemost
JT  - Research and practice in thrombosis and haemostasis
JID - 101703775
PMC - PMC9663319
OTO - NOTNLM
OT  - antibody
OT  - blood coagulation factors
OT  - clinical trial
OT  - hemophilia A
OT  - hemostasis
EDAT- 2022/11/19 06:00
MHDA- 2022/11/19 06:01
PMCR- 2022/11/14
CRDT- 2022/11/18 02:07
PHST- 2022/03/24 00:00 [received]
PHST- 2022/10/07 00:00 [revised]
PHST- 2022/10/09 00:00 [accepted]
PHST- 2022/11/18 02:07 [entrez]
PHST- 2022/11/19 06:00 [pubmed]
PHST- 2022/11/19 06:01 [medline]
PHST- 2022/11/14 00:00 [pmc-release]
AID - S2475-0379(22)02432-3 [pii]
AID - RTH212837 [pii]
AID - 10.1002/rth2.12837 [doi]
PST - epublish
SO  - Res Pract Thromb Haemost. 2022 Nov 14;6(8):e12837. doi: 10.1002/rth2.12837. 
      eCollection 2022 Nov.