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Journal Articles

Quality of life and functional improvements with efanesoctocog alfa in patients with moderate-to-severe Hemophilia A: A real-world survey

Maissaa Janbain, Miguel Escobar, Manuel Carcao, Nathan Ball, Ella Morton, Duygu Bozkaya, Sylvie Bozzi, Nicole Tsao, Andrew Wilson

Blood (2025) 146 (Supplement 1): 4846.

https://doi.org/10.1182/blood-2025-4846

Published: 2025

Journal Articles

Emicizumab prophylaxis in pups and mtps with severe haemophilia: The pednet real world experience in 80 infants

Marloes de Kovel, Gili Kenet, Jayashree Motwani, Nadine G. Andersson, Jan Blanty, Giancarlo Castaman, Manuel Carcao, Carmen Escuriola-Ettingshausen, Chris Königs, Christoph Male, Beatrice Nolan, Martin Olivieri, Caroline Oudot, Eleni Pergantou, Susanna Ranta, Ester Zapotocka, Kathelijn Fischer

Blood (2025) 146 (Supplement 1): 305.

https://doi.org/10.1182/blood-2025-305

Published: 2025

Journal Articles

Effectiveness of sirolimus for pediatric autoimmune cytopenias and factors associated with clinical response

Maggie McNeill, Camilla Cellarius, Nethmi Illamperuma, Rachelle Zipper, Clarisse Carcao, Jillian Baker, Sheila Butchart, Michelle Fantauzzi, Melanie Kirby, Caroline Malcolmson, Reena Pabari, Diandra Rollan, Manuel Carcao

Blood (2025) 146 (Supplement 1): 4810.

https://doi.org/10.1182/blood-2025-4810

Published: 2025

Journal Articles

Morbidity and mortality in 128 otherwise healthy Canadian children with severe iron deficiency anemia in the setting of ongoing bottle use and excessive cow's milk intake: A massive preventable problem demanding action

Rachelle Zipper, Clarisse Carcao, Maggie McNeill, Nethmi Illamperuma, Camilla Cellarius, Jillian Baker, Sheila Butchart, Michelle Fantauzzi, Melanie Kirby-Allen, Hannah Leu, Caroline Malcolmson, Reena Pabari, Renee Potashner, Diandra Rollan, Manuel Carcao

Blood (2025) 146 (Supplement 1): 2903.

https://doi.org/10.1182/blood-2025-2903

Published: 2025

Journal Articles

Real-World Experience of Switching to Prophylactic Efanesoctocog Alfa in Patients with Moderate and Severe Hemophilia a: An Analysis of the Adelphi Hemophilia Wave III Disease Specific Programme

Maissaa Janbain, Miguel A. Escobar, Manuel Carcao, Andrew Wilson, Jennifer Dumont, Nathan Ball, Ella Morton, Duygu Bozkaya, Anne-Laure Tardy, Sylvie Bozzi

Blood (2024) 144 (Supplement 1): 131.

https://doi.org/10.1182/blood-2024-193764

Published: 2024

Journal Articles

A Longitudinal Examination of the Quality of Life of Persons with Hemophilia a Switching from Intravenous Recombinant Factor VIII Concentrate to Subcutaneous Non-FVIII Replacement Therapy

Roxy O'Rourke, Vanessa Bouskill, Cindy Wakefield, Manuel Carcao, Audrey Abad, Victor Blanchette, Caroline Malcolmson

Blood (2024) 144 (Supplement 1): 7698.

https://doi.org/10.1182/blood-2024-207794

Published: 2024

Journal Articles

Non-Factor Therapy Reduces Burden of Care for Caregivers of Children with Severe Hemophilia a: A Longitudinal Cohort Study Using the Hemophilia Family Impact Tool (H-FIT v1.1)

Roxy O'Rourke, Vanessa Bouskill, Cindy Wakefield, Manuel Carcao, Audrey Abad, Victor Blanchette, Caroline Malcolmson

Blood (2024) 144 (Supplement 1): 7709.

https://doi.org/10.1182/blood-2024-211188

Published: 2024

Journal Articles

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Blood (2023) 142 (Supplement 1): 5480.

https://doi.org/10.1182/blood-2023-181299

Published: 2023

Journal Articles

Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children with Severe Hemophilia A in the XTEND-Kids Phase 3 Study

Lynn Malec, Amy Dunn, Manuel Carcao, Bulent Zulfikar, Ruben Berrueco, Simon Brown, Umer Khan, Sriya Gunawardena, Graham Neill, Lydia Abad-Franch, Linda Bystrická, Elena Santagostino, Sophie Susen

Blood (2023) 142 (Supplement 1): 3993.

https://doi.org/10.1182/blood-2023-188739

Published: 2023

Journal Articles

Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated Patients with Hemophilia A from the XTEND-Kids Phase 3 Clinical Trial

Lynn Malec, Manuel Carcao, Mary Mathias, Nana Kragh, Jennifer Dumont, Annemieke Willemze, Linda Bystrická, Amanda Wilson, Dana Dibenedetti

Blood (2023) 142 (Supplement 1): 507.

https://doi.org/10.1182/blood-2023-181973

Published: 2023

Journal Articles

Recombinant factor VIII Fc fusion protein for first-time immune tolerance induction: final results of the verITI-8 study

Lynn Malec, An Van Damme, Anthony K. C. Chan, Mariya Spasova, Nisha Jain, Charlotte Sensinger, Jennifer Dumont, Stefan Lethagen, Manuel Carcao, Flora Peyvandi

Blood (2023) 141 (16): 1982–1989.

https://doi.org/10.1182/blood.2022017780

Published: 2023

Includes: Supplemental data

Journal Articles

Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

Amy L. Dunn, Yesim Dargaud, Yasmina L. Abajas, Manuel Carcao, Giancarlo Castaman, Adam Giermasz, Cédric Hermans, Magdalena D. Lewandowska, Johnny Mahlangu, Shannon L. Meeks, Wolfgang A. Miesbach, Michael Recht, Vanessa Salinas, Tammuella Chrisentery-Singleton, Hongying Wang, Ian S Mitchell, Guy Young

Blood (2022) 140 (Supplement 1): 8485–8488.

https://doi.org/10.1182/blood-2022-157343

Published: 2022

Journal Articles

Long-Term Follow-up of Patients with Congenital Thrombotic Thrombocytopenia Purpura Receiving Plasma-Derived Factor VIII Containing (Koate®)

Tammuella Chrisentery-Singleton, Lisa N Boggio, Manuel Carcao, Sami Ibrahimi, Osman Khan, Arash Mahajerin, Anita Rajasekhar, Vivek Sharma, MacGregor Steele, Marcela Torres, Shannon L Carpenter

Blood (2022) 140 (Supplement 1): 5644–5646.

https://doi.org/10.1182/blood-2022-162506

Published: 2022

Journal Articles

A Real-World Population Pharmacokinetic (PK) Study of Two Recombinant Factor VIII (rFVIII) Concentrates in Boys with Severe Hemophilia a without Inhibitors

Viviane Grassmann, Dagmar M Hajducek, Andrea N Edginton, Vanessa Bouskill, Christine Brown, Manuel Carcao, David Lillicrap, Roxy H O'Rourke, Roona Sinha, Jayson Stoffman, Cindy Wakefield, Margaret L. Rand, Victor Blanchette

Blood (2022) 140 (Supplement 1): 8453–8455.

https://doi.org/10.1182/blood-2022-160289

Published: 2022

Journal Articles

Unravelling the Effect of Blood Type on FVIII:C Levels and Response to DDAVP in 20 Males with a Single Genotype ( Twillingate Variant) Causing Mild Hemophilia Α

Caroline Malcolmson, Michael Shu, Vanessa Bouskill, Ann Marie Stain, Cindy Wakefield, Victor Blanchette, Manuel Carcao

Blood (2022) 140 (Supplement 1): 5588–5590.

https://doi.org/10.1182/blood-2022-169675

Published: 2022

Journal Articles

Moderate-intensity aerobic exercise vs desmopressin in adolescent males with mild hemophilia A: a randomized trial

Riten Kumar, Amy L. Dunn, Jane E. Schneiderman, Anne Gonzales, Vanessa Bouskill, Pamela Widener, Joseph Stanek, Fred G. Pluthero, Amanda Waller, Cristina Tarango, Sanjay Ahuja, Bryce A. Kerlin, Walter H. A. Kahr, Margaret L. Rand, David Lillicrap, Manuel Carcao

Blood (2022) 140 (10): 1156–1166.

https://doi.org/10.1182/blood.2022016146

Published: 2022

Includes: Supplemental data

Journal Articles

First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results

Christoph Königs, Margareth C. Ozelo, Amy Dunn, Roshni Kulkarni, Beatrice Nolan, Simon A. Brown, Michele Schiavulli, Sriya Gunawardena, Sutirtha Mukhopadhyay, Deepthi Jayawardene, Bent Winding, Manuel Carcao

Blood (2022) 139 (26): 3699–3707.

https://doi.org/10.1182/blood.2021013563

Published: 2022

Includes: Supplemental data

Journal Articles

Efficacy of rFVIIIFc for First-Time Immune Tolerance Induction (ITI) Therapy: Final Results from the Global, Prospective VerITI-8 Study

Lynn Malec, An Van Damme, Anthony Chan, Mariya Spasova, Nisha Jain, Charlotte Sensinger, Jennifer Dumont, Stefan Lethagen, Manuel Carcao, Flora Peyvandi

Blood (2021) 138 (Supplement 2): LBA-5.

https://doi.org/10.1182/blood-2021-155156

Published: 2021

Journal Articles

Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age

Blood (2021) 138 (Supplement 1): 3195.

https://doi.org/10.1182/blood-2021-147434

Published: 2021

Journal Articles

Clingen Coagulation Factor Deficiency Variant Curation Expert Panel: Meeting the Need for Recommendations to Curate Variants in the Coagulation Factor Genes

Shruthi Mohan, PhD, Kristy Lee, MS, CGC, Manuel Carcao, Bhavya S Doshi, MD, Kate Downes, PhD, Geoff Kemball-Cook, PhD, Frank Leebeek, MD PhD, Jamie McCreery, MS, CGC, Connie H. Miller, PhD, Amanda B Payne, MPH,PhD, Valerie Trapp-Stamborski, PhD, Barbara A. Konkle, MD, Keith Gomez, MBBS, PhD MRCP, FRCPath

Blood (2019) 134 (Supplement_1): 5794.

https://doi.org/10.1182/blood-2019-124842

Published: 2019

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Quality of life and functional improvements with efanesoctocog alfa in patients with moderate-to-severe Hemophilia A: A real-world survey

Emicizumab prophylaxis in pups and mtps with severe haemophilia: The pednet real world experience in 80 infants

Effectiveness of sirolimus for pediatric autoimmune cytopenias and factors associated with clinical response

Morbidity and mortality in 128 otherwise healthy Canadian children with severe iron deficiency anemia in the setting of ongoing bottle use and excessive cow's milk intake: A massive preventable problem demanding action

Real-World Experience of Switching to Prophylactic Efanesoctocog Alfa in Patients with Moderate and Severe Hemophilia a: An Analysis of the Adelphi Hemophilia Wave III Disease Specific Programme

A Longitudinal Examination of the Quality of Life of Persons with Hemophilia a Switching from Intravenous Recombinant Factor VIII Concentrate to Subcutaneous Non-FVIII Replacement Therapy

Non-Factor Therapy Reduces Burden of Care for Caregivers of Children with Severe Hemophilia a: A Longitudinal Cohort Study Using the Hemophilia Family Impact Tool (H-FIT v1.1)

Low Rebleed Incidence with Eptacog Beta Treatment through 24 and 48 Hours in Children (<12 years) with Hemophilia a or B with Inhibitors

Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children with Severe Hemophilia A in the XTEND-Kids Phase 3 Study

Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated Patients with Hemophilia A from the XTEND-Kids Phase 3 Clinical Trial

Recombinant factor VIII Fc fusion protein for first-time immune tolerance induction: final results of the verITI-8 study

Eptacog Beta (rFVIIa) Has a Low Incidence of Spontaneous Rebleeding through 24 and 48 Hours in Adult and Adolescent Patients with Hemophilia A or B with Inhibitors

Long-Term Follow-up of Patients with Congenital Thrombotic Thrombocytopenia Purpura Receiving Plasma-Derived Factor VIII Containing (Koate®)

A Real-World Population Pharmacokinetic (PK) Study of Two Recombinant Factor VIII (rFVIII) Concentrates in Boys with Severe Hemophilia a without Inhibitors

Unravelling the Effect of Blood Type on FVIII:C Levels and Response to DDAVP in 20 Males with a Single Genotype ( Twillingate Variant) Causing Mild Hemophilia Α

Moderate-intensity aerobic exercise vs desmopressin in adolescent males with mild hemophilia A: a randomized trial

First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results

Efficacy of rFVIIIFc for First-Time Immune Tolerance Induction (ITI) Therapy: Final Results from the Global, Prospective VerITI-8 Study

Eptacog Beta Efficacy in Children and Adolescents with Hemophilia A or B and Inhibitors: Subset Analysis Suggests Improved Caregiver Capacity to Assess Bleeding Episode Resolution with Subject Age

Clingen Coagulation Factor Deficiency Variant Curation Expert Panel: Meeting the Need for Recommendations to Curate Variants in the Coagulation Factor Genes

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