App-Quantinova.ai

A prospective analysis of breakthrough haemolysis in two Phase 3 randomised studies of ravulizumab (ALXN1210) versus eculizumab in adults with paroxysmal nocturnal haemoglobinuria

R.A. Brodsky - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Nephrology

Paroxysmal nocturnal hemoglobinuria

Eculizumab

The BELIEVE Trial: Results of a Phase 3, Randomised, Double‐Blind, Placebo‐Controlled Study of Luspatercept in Adult β‐Thalassaemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

J. Porter - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Transfusion‐dependent β‐thalassaemia

luspatercept

A Phase 1 study to determine maximum tolerated dose of sublingual fentanyl in combination with oral oxycodone for hospital management of an acute painful crisis in adolescents and adults with sickle cell disease

P. Telfer - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Gastroenterology (GU)

Acute chest syndrome

Oxycodone

Characterising Red Cell‐Derived Vesicles In Sickle Cell Disease

D. Drizou - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Central Nervous System (CNS)

Sickle cell anemia

N/A

An observational study to evaluate the routine management, healthcare resource use and outcomes for patients with transfusion‐dependent β‐thalassaemia treated in the United Kingdom: an interim analysis

K. Ryan - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Genetic Disorder

Transfusion‐dependent β‐thalassaemia

Deferoxamine

Whole genome sequencing for the investigation of rare anaemias: Challenges and real‐world outcomes

C. Brierley - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

N/A

Peer to peer mentoring for patients with Sickle Cell Disease – interim analysis of results from a pilot programme in East London

J. Okochi - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

N/A

Psychological need in sickle cell disease: evaluation of psychological functioning during the annual review process

J. Love - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Musculoskeletal

Sickle cell anemia

N/A

Glucophosphate isomerase deficiency identified by next‐generation sequencing

S.M. Vora - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Gastroenterology (GU)

Glucophosphate isomerase deficiency

N/A

Addressing the need for an external quality assessment for next generation sequencing (NGS) in the diagnosis of rare inherited anaemias.

N. Roy - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Genetic Disorder

Velocardiofacial syndrome

N/A

Development and validation of the Satisfaction with Treatment for Pain Questionnaire (STPQ) among people with sickle cell disease

P. Telfer - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

N/A

Evaluation of a weekly exercise group for people living with sickle cell disease

R. McLoughlin - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Musculoskeletal

Sickle cell anemia

N/A

Tociluzimab for the Management of Recurrent Resistant Post Transfusion Hyperhaemolysis in Sickle Cell Disease

L. Linpower - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Pulmonary/Respiratory Diseases

Acute chest syndrome

Tocilizumab

Pregnancy Outcomes in Patients with Sickle Cell Syndromes Managed Antenatally with Exchange Transfusions: a single centre analysis

E. Vlachodimitropoulou - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

Allopurinol

Efficacy of eculizumab in paediatric patients with paroxysmal nocturnal haemoglobinuria in the International PNH Registry

. Urbano-Ispizua - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Paroxysmal nocturnal hemoglobinuria

Eculizumab

Prognostic value of clone size in paroxysmal nocturnal hemoglobinuria (PNH) for thrombotic events in untreated patients in the International PNH Registry

R. Peffault De Latour - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Cardiovascular (CVS)

Paroxysmal nocturnal hemoglobinuria

N/A

Patients lost to follow‐up in the East London Newborn Sickle Cell Cohort Study

C. Sangarappillai - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Blood/haematological Diseases

Sickle cell anemia

N/A

Fat Embolism Syndrome in Sickle Cell Disease: Current State and Future Prospects

D.A. Tsitsikas - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Nephrology

Fat embolism syndrome

N/A

Prevalence of small PNH clones and their prognostic significance in patients tested for unusual indications: a single center experience

B. Fattizzo - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Cardiovascular (CVS)

Paroxysmal nocturnal hemoglobinuria

N/A

Fat Embolism Syndrome in a patient with homozygous Sickle Cell Disease in association with Influenza A

R. Mullally - British Society for Hematology (BSH) , 2019

Therapeutic Area

Disease

Drug/Treatment

Musculoskeletal

Fat embolism syndrome

Methylprednisolone

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