A K Chan

Clinical Outcomes of Efanesoctocog Alfa Prophylaxis in Children With Severe Haemophilia A: Interim Data From XTEND‐ed

Lynn Malec, Beatrice Nolan, A K Chan, Manuela Albisetti et autres

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Defining Roles in Pediatric Palliative Care: Perspectives From Oncology and Palliative Care Teams

Leeat Granek, Lori S. Wiener, Stephanie Veldhuijzen van Zanten, Donna Johnston et autres

BACKGROUND: Early integration of pediatric palliative care (PPC) is associated with improved symptom management, quality of life, and healthcare utilization for children with cancer. Despite this, variation persists in how PPC is understood, operationalized, and integrated within pediatric oncology programs. In particular, …

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Case Report: Atypical presentations of neonatal and infantile hemophilia B

Arielle Locke, Nasrin Samji, Mihir Bhatt, Kay Decker et autres

Hemophilia B is an X-linked recessive bleeding disorder caused by deficiency or dysfunction of factor IX. It typically presents with spontaneous or trauma-induced bleeding, hemarthrosis, and soft tissue hematomas. We report three cases-two severe neonatal and one moderate infantile case-with atypical presentations: …

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Mim8 Bispecific Antibody Prophylaxis in Hemophilia A with or without Inhibitors

Maria Elisa Mancuso, A K Chan, Chandrakala Shanmukhaiah, Chuhl Joo Lyu et autres

BACKGROUND: Mim8 (denecimig), a bispecific antibody mimicking activated factor VIII, was developed for bleeding prophylaxis in patients with hemophilia A with or without factor VIII inhibitors. METHODS: In this phase 3, randomized trial, we assigned patients 12 years of age or older …

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Damoctocog alfa pegol in patients aged 7 to<12; an interim analysis of the Alfa-PROTECT extension

A K Chan, M C Ozelo, Matteo Luciani, Heidi Glosli et autres

Introduction The safety and efficacy of damoctocog alfa pegol for the prevention and treatment of bleeding in previously treated children with severe hemophilia A has been re-examined in the Alfa-PROTECT trial (NCT05147662) and its 18-month extension. Here, we aim to evaluate the …

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Minor Surgeries Outcomes with Efanesoctocog Alfa: 4 Years’ Experience in the XTEND Clinical Program

Pratima Chowdary, Azusa Nagao, J Oldenburg, L Malec et autres

Introduction Efanesoctocog alfa is a first-in-class high sustained factor VIII replacement therapy that overcomes the von Willebrand factor-imposed half-life ceiling. In XTEND-1 (NCT04161495) and XTEND-Kids (NCT04759131), once-weekly efanesoctocog alfa was well tolerated and highly efficacious for perioperative management of bleeding in patients …

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