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Journal Articles

Predicting Vaso-Occlusive Crises in Sickle Cell Disease through Digital, Longitudinal Tracking of Wearable Metrics and Patient-Reported Outcomes

Kim Summers, Orlando Agrippa, Sanne Lugthart, Kofi Anie, Paul Telfer

Blood (2023) 142 (Supplement 1): 1059.

https://doi.org/10.1182/blood-2023-181407

Published: 2023

Journal Articles

Over 4 Years of Safety and Efficacy with Voxelotor Treatment for Patients with Sickle Cell Disease: Updated Results from an Open-Label Extension of the Phase 3 HOPE Trial

Maureen Okam Achebe, Hoda MA Hassab, Salam Al-Kindi, Clark Brown, Paul Telfer, Bart J. Biemond, Thokozeni Lipato, Mark Davis, Sarah Gray, Victor R. Gordeuk

Blood (2023) 142 (Supplement 1): 2527.

https://doi.org/10.1182/blood-2023-188616

Published: 2023

Journal Articles

Real-World Outcomes and Digitally Monitored Quality of Life in Voxelotor-Treated Patients with Sickle Cell Disease

Kim Summers, Orlando Agrippa, Oliver Shastri, Seraj Sharif, Kofi Anie, Paul Telfer, Sanne Lugthart

Blood (2023) 142 (Supplement 1): 2383.

https://doi.org/10.1182/blood-2023-181426

Published: 2023

Journal Articles

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2023) 142 (Supplement 1): 1052.

https://doi.org/10.1182/blood-2023-190139

Published: 2023

Journal Articles

Severe Acute Complications of Sickle Cell Disease in Two Expert Referral Centers (UK and Italy): Natural History Studies Highlight Ongoing Unmet Need for Effective Disease Modifying or Curative Therapies

Raffaella Colombatti, Cynthia Sangarappillai, Muriel Soriano, Jonathan Bestwick, Will Hann, Karolina Wieczorek, Vania Munaretto, Beatrice Coppadoro, Giulia Reggiani, Laura Sainati, Anjulika Chawla, Charu Puri-Sharma, Courtney Walls, Sanjeev Kommera, Paul Telfer

Blood (2021) 138 (Supplement 1): 3093.

https://doi.org/10.1182/blood-2021-151407

Published: 2021

Journal Articles

Long-Term Safety and Efficacy of Voxelotor for Patients with Sickle Cell Disease: Results from an Open-Label Extension of the Phase 3 HOPE Trial

Maureen Achebe, Hoda Hassab, Salam Alkindi, R. Clark Clark Brown, Paul Telfer, Bart J. Biemond, Victor R. Gordeuk, Thokozeni Lipato, Margaret Tonda, Sarah Gray, Joanna Howard

Blood (2021) 138 (Supplement 1): 3114.

https://doi.org/10.1182/blood-2021-153582

Published: 2021

Journal Articles

Higher Hemoglobin Levels Achieved with Voxelotor Are Associated with Lower Vaso-occlusive Crisis Incidence: 72-Week Analysis from the HOPE Study

Elliott Vichinsky, MD, Victor R. Gordeuk, MD, Paul Telfer, DM, FRCP, Adlette Inati, MD FAAP, Margaret Tonda, PharmD, Sarah Gray, PhD, Irene Agodoa, MD, Kenneth I. Ataga, MD

Blood (2020) 136 (Supplement 1): 31–32.

https://doi.org/10.1182/blood-2020-140863

Published: 2020

Journal Articles

Improvement in the Clinical Global Impression of Change with Voxelotor in Patients with Sickle Cell Disease in the Phase 3 HOPE Trial

Wally R Smith, MD, Jane S. Hankins, MD MS, Miguel R Abboud, MD, Ze Cong, PhD, Jonathan Sorof, MD, Sarah Gray, PhD, Carolyn Hoppe, MD, Paul Telfer, DM, FRCP

Blood (2020) 136 (Supplement 1): 5–6.

https://doi.org/10.1182/blood-2020-137121

Published: 2020

Journal Articles

Routine Management, Healthcare Resource Use and Patient/Caregiver-Reported Outcomes of Patients with Transfusion-Dependent β-Thalassaemia in the United Kingdom: A Mixed Methods Observational Study

Farrukh Shah, MDMBBS,FRCPath, Paul Telfer, DM, FRCP, Mark Velangi, Shivan Pancham, Robert Wynn, Sally Pollard, Elizabeth Chalmers, Jonathan Kell, MD, Joe Hickey, PhD, Clark Paramore, MPH, Minesh Jobanputra, MD, Kate Ryan, MD

Blood (2019) 134 (Supplement_1): 3550.

https://doi.org/10.1182/blood-2019-129930

Published: 2019

Journal Articles

Incidence of Vaso-Occlusive Crisis Does Not Increase with Achieving Higher Hemoglobin Levels on Voxelotor Treatment or after Discontinuation: Analyses of the HOPE Study

Elliott P. Vichinsky, MD, Paul Telfer, DM, FRCP, Adlette Inati, MD, Margaret Tonda, PharmD, Barbara Tong, PhD, Irene Agodoa, MD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Kenneth I. Ataga, MD

Blood (2019) 134 (Supplement_1): 2313.

https://doi.org/10.1182/blood-2019-129026

Published: 2019

Journal Articles

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease

Jo Howard, Claire Jane Hemmaway, Paul Telfer, D. Mark Layton, John Porter, Moji Awogbade, Timothy Mant, Daniel D. Gretler, Kobina Dufu, Athiwat Hutchaleelaha, Mira Patel, Vincent Siu, Sandra Dixon, Noel Landsman, Margaret Tonda, Joshua Lehrer-Graiwer

Blood (2019) 133 (17): 1865–1875.

https://doi.org/10.1182/blood-2018-08-868893

Published: 2019

Journal Articles

Hydroxyurea Therapy Does Not Impact Current Survival Estimates in the East London Sickle Cell Newborn Cohort Study

Paul Telfer, Banu Kaya, MD, Dimitris A. Tsitsikas, Filipa Barroso, MD, Cynthia Sangarappillai, BSc

Blood (2018) 132 (Supplement 1): 3655.

https://doi.org/10.1182/blood-2018-99-116544

Published: 2018

Journal Articles

Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a Placebo-Controlled Randomized Study Evaluating Voxelotor (GBT440) in Adults and Adolescents with Sickle Cell Disease

Blood (2018) 132 (Supplement 1): 505.

https://doi.org/10.1182/blood-2018-99-118508

Published: 2018

Journal Articles

Impact of GBT440 on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease: A Patient Case Report

Paul Telfer, FRCPath, MD, Josh Lehrer, MD MPhil, FACC, Irene Agodoa, MD, Marzena Jurek, MS, Kathleen M Fox, PhD, Laurie Burke, BPharm, MPH, Noel Landsman, Dip.Nur (SA), Timothy Mant, MB, FFPM, FRCP

Blood (2017) 130 (Supplement 1): 4779.

https://doi.org/10.1182/blood.V130.Suppl_1.4779.4779

Published: 2017

Journal Articles

Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor

Josh Lehrer-Graiwer, MDMPhil, FACC, Jo Howard, MB, BChir, Claire Jane Hemmaway, MD, Moji Awogbade, Paul Telfer, FRCPath,MD, Mark Layton, MB, FRCP, John B. Porter, MD, Timothy Mant, MB, FFPM, FRCP, Kobina Dufu, PhD, Athiwat Hutchaleelaha, PhD, Donna Oksenberg, PhD, Mira Patel, PhD, Margaret Eleanor Tonda, PharmD, Kenneth Bridges, MD, Eleanor Ramos, MD

Blood (2016) 128 (22): 2488.

https://doi.org/10.1182/blood.V128.22.2488.2488

Published: 2016

Journal Articles

Haploidentical BMT with a Post-Infusion of Stem Cells Cyclophosphamide Approach Is Feasible and Leads to a High Rate of Donor Engraftment in Haemoglobinopathies Allowing Universal Application of Transplantation

Josu de la Fuente, PhD FRCP, FRCPI, FRCPCH, FRCPath, Farah O'Boyle, PhD, Yvonne Harrington, Anne Bradshaw, Sandra Hing, PhD, Subarna Chakravorty, PhD FRCPath, Leena Karnik, MD FRCPath, MBBS,, Helen New, PhDFRCPath, Richard Szydlo, PhD, Baba PD Inusa, MBBS, FMCPaed, FRCPCH, MRCP, DCP, Paul Telfer, FRCPath,MD

Blood (2015) 126 (23): 4317.

https://doi.org/10.1182/blood.V126.23.4317.4317

Published: 2015

Journal Articles

GBT440, a Potent Anti-Sickling Hemoglobin Modifier Reduces Hemolysis, Improves Anemia and Nearly Eliminates Sickle Cells in Peripheral Blood of Patients with Sickle Cell Disease

Josh Lehrer-Graiwer, MDMPhil, FACC, Jo Howard, MD, Claire Jane Hemmaway, MD, Moji Awogbade, Paul Telfer, FRCPath,MD, Mark Layton, MB, FRCP, Timothy Mant, MB, FFPM, FRCP, Kobina Dufu, PhD, Athiwat Hutchaleelaha, PhD, Teri Koller, MBA, Donna Oksenberg, PhD, Mira Patel, PhD, Eleanor Ramos, MD

Blood (2015) 126 (23): 542.

https://doi.org/10.1182/blood.V126.23.542.542

Published: 2015

Journal Articles

A Multicentre Study of Environmental Factors on the Severity of Sickle Cell Disease

Sanjay Tewari, MBBS DCH MRCPCH, Fred Piel, PhD, Valentine Brousse, MD, Baba PD Inusa, MD FRCPCH, DCP, Paul Telfer, MD, Stephan Menzel, MD, Mariane De Montalembert, Kate Gardner, Gary Fuller, Swee Lay Thein, DSc, FRCP, FMedSci, MB, BS, FRCP, David Rees, FRCPCH

Blood (2014) 124 (21): 4841.

https://doi.org/10.1182/blood.V124.21.4841.4841

Published: 2014

Journal Articles

Disease-Related Factors and Family Functioning As Predictors of Executive Functioning in Preschool Children with Sickle Cell Anaemia: A Preliminary Analysis

Michelle Downes, Msc, Michelle de Haan, PhD, Fenella J. Kirkham, MD, Banu Kaya, MD, Paul Telfer, MD

Blood (2014) 124 (21): 4941.

https://doi.org/10.1182/blood.V124.21.4941.4941

Published: 2014

Journal Articles

Inadequate Long-Term Control Of Transfusion Iron Overload In Children With Sickle Cell Disease Chelated With Deferasirox

Eva Tsouana, Banu Kaya, Nimze Gadong, Claire Hemmaway, Heather Newell, Andrea Simmons, Simon Whitmarsh, Paul Telfer, MA, DM, BM, BCh, FRCP, FRCPath

Blood (2013) 122 (21): 998.

https://doi.org/10.1182/blood.V122.21.998.998

Published: 2013

NARROW

Predicting Vaso-Occlusive Crises in Sickle Cell Disease through Digital, Longitudinal Tracking of Wearable Metrics and Patient-Reported Outcomes

Over 4 Years of Safety and Efficacy with Voxelotor Treatment for Patients with Sickle Cell Disease: Updated Results from an Open-Label Extension of the Phase 3 HOPE Trial

Real-World Outcomes and Digitally Monitored Quality of Life in Voxelotor-Treated Patients with Sickle Cell Disease

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Severe Acute Complications of Sickle Cell Disease in Two Expert Referral Centers (UK and Italy): Natural History Studies Highlight Ongoing Unmet Need for Effective Disease Modifying or Curative Therapies

Long-Term Safety and Efficacy of Voxelotor for Patients with Sickle Cell Disease: Results from an Open-Label Extension of the Phase 3 HOPE Trial

Higher Hemoglobin Levels Achieved with Voxelotor Are Associated with Lower Vaso-occlusive Crisis Incidence: 72-Week Analysis from the HOPE Study

Improvement in the Clinical Global Impression of Change with Voxelotor in Patients with Sickle Cell Disease in the Phase 3 HOPE Trial

Routine Management, Healthcare Resource Use and Patient/Caregiver-Reported Outcomes of Patients with Transfusion-Dependent β-Thalassaemia in the United Kingdom: A Mixed Methods Observational Study

Incidence of Vaso-Occlusive Crisis Does Not Increase with Achieving Higher Hemoglobin Levels on Voxelotor Treatment or after Discontinuation: Analyses of the HOPE Study

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease

Hydroxyurea Therapy Does Not Impact Current Survival Estimates in the East London Sickle Cell Newborn Cohort Study

Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a Placebo-Controlled Randomized Study Evaluating Voxelotor (GBT440) in Adults and Adolescents with Sickle Cell Disease

Impact of GBT440 on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease: A Patient Case Report

Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor

Haploidentical BMT with a Post-Infusion of Stem Cells Cyclophosphamide Approach Is Feasible and Leads to a High Rate of Donor Engraftment in Haemoglobinopathies Allowing Universal Application of Transplantation

GBT440, a Potent Anti-Sickling Hemoglobin Modifier Reduces Hemolysis, Improves Anemia and Nearly Eliminates Sickle Cells in Peripheral Blood of Patients with Sickle Cell Disease

A Multicentre Study of Environmental Factors on the Severity of Sickle Cell Disease

Disease-Related Factors and Family Functioning As Predictors of Executive Functioning in Preschool Children with Sickle Cell Anaemia: A Preliminary Analysis

Inadequate Long-Term Control Of Transfusion Iron Overload In Children With Sickle Cell Disease Chelated With Deferasirox

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