Publications: DR Paul Telfer
(
2025
)
.
Can health inequalities in sickle cell disease be addressed through novel therapies?
.
HemaSphere
vol.
9
,
(
7
)
Article
e70175
,
(
2025
)
.
PT38 Assessing the Impact of Digital Ecosystem Engagement on Outcomes in Sickle Cell Disease (SCD) Patients
.
Value in Health
vol.
28
,
(
6
)
S361
-
S361
.
(
2025
)
.
Durable Clinical Benefits in Severe Sickle Cell Disease with Exagamglogene Autotemcel
.
Transplantation and Cellular Therapy
vol.
31
,
(
2
)
S20
-
S21
.
(
2024
)
.
A Virtual Hospital Approach in Sickle Cell Disease | Remote Biometric, Quality of Life, and Hospitalisations Monitoring
.
Blood
vol.
144
,
(
Supplement 1
)
5072
-
5072
.
(
2024
)
.
Comparison of Sickle Cell Disease Patient Experiences in Developed and Developing Countries: Insights from UK and India Patient Cohorts
.
Blood
vol.
144
,
(
Supplement 1
)
5071
-
5071
.
(
2024
)
.
Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease
.
Blood
vol.
144
,
(
Supplement 1
)
4954
-
4954
.
(
2024
)
.
Enhanced Artificial Intelligence (AI)-Driven Prediction of Vaso-Occlusive Crises in Sickle Cell Disease: Precision through Advanced Machine-Learning Frameworks and Digital Remote Monitoring
.
Blood
vol.
144
,
(
Supplement 1
)
522
-
522
.
(
2024
)
.
Over 4 Years of Safety and Efficacy with Voxelotor in Patients with Sickle Cell Disease
.
Journal of Sickle Cell Disease
vol.
1
,
(
Supplement_1
)
Article
yoae002.008
,
(
2024
)
.
Tolérance et efficacité de voxelotor sur une période de plus de 4 ans chez les patients atteints de drépanocytose : résultats actualisés d’une étude d’extension en ouvert de l’essai de phase 3 HOPE
.
La Revue de Médecine Interne
vol.
45
,
A130
-
A131
.
(
2024
)
.
Exagamglogene Autotemcel for Severe Sickle Cell Disease
.
New England Journal of Medicine
vol.
390
,
(
18
)
1649
-
1662
.
(
2024
)
.
The acute pain crisis in sickle cell disease: What can be done to improve outcomes?
.
Blood Reviews
vol.
65
,
Article
101194
,
101194
-
101194
.
(
2024
)
.
Elimination of Vaso-Occlusive Crises after Exagamglogene Autotemcel in Patients with Severe Sickle Cell Disease
.
Transplantation and Cellular Therapy
vol.
30
,
(
2
)
S234
-
S235
.
(
2024
)
.
Haematopoietic stem cell health in sickle cell disease and its implications for stem cell therapies and secondary haematological disorders
.
Blood Reviews
vol.
63
,
Article
101137
,
101137
-
101137
.
(
2023
)
.
Exagamglogene Autotemcel for Severe Sickle Cell Disease
.
Blood
vol.
142
,
(
Supplement 1
)
1052
-
1052
.
(
2023
)
.
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
.
Blood
vol.
142
,
(
Supplement 1
)
2527
-
2527
.
(
2023
)
.
Predicting Vaso-Occlusive Crises in Sickle Cell Disease through Digital, Longitudinal Tracking of Wearable Metrics and Patient-Reported Outcomes
.
Blood
vol.
142
,
(
Supplement 1
)
1059
-
1059
.
(
2023
)
.
Real-World Outcomes and Digitally Monitored Quality of Life in Voxelotor-Treated Patients with Sickle Cell Disease
.
Blood
vol.
142
,
(
Supplement 1
)
2383
-
2383
.
(
2023
)
.
Efficacy, safety, and pharmacokinetics of a new, ready-to-use, liquid hydroxyurea in children with sickle cell anemia
.
Blood Advances
vol.
7
,
(
16
)
4319
-
4322
.
(
2023
)
.
P1428: PREDICTORS OF VOC RATE DURING LONG-TERM FOLLOW-UP OF PATIENTS WITH HBSS IN A NEWBORN COHORT STUDY
.
HemaSphere
vol.
7
,
(
S3
)
e7763412
-
e7763412
.
(
2023
)
.
P1441: REAL-WORLD OUTCOMES AND DIGITALLY MONITORED QUALITY OF LIFE IN CRIZANLIZUMAB-TREATED PATIENTS WITH SICKLE CELL DISEASE
.
HemaSphere
vol.
7
,
(
S3
)
e25264f3
-
e25264f3
.
(
2023
)
.
P1448: CHARACTERISATION OF QUALITY OF LIFE-LINKED PATIENT-REPORTED OUTCOMES THROUGH A DIGITAL AND CONTINUOUS REMOTE MONITORING ECOSYSTEM IN SICKLE CELL DISEASE
.
HemaSphere
vol.
7
,
(
S3
)
e53344bb
-
e53344bb
.
(
2023
)
.
Direct correction of haemoglobin E β-thalassaemia using base editors
.
Nature Communications
vol.
14
,
(
1
)
Article
2238
,
(
2023
)
.
5554869 ASSOCIATION BETWEEN HEMOGLOBIN LEVELS AND END-ORGAN DAMAGE IN SICKLE CELL DISEASE: A RETROSPECTIVE LINKED PRIMARY AND SECONDARY CARE DATABASE ANALYSIS IN ENGLAND
.
HemaSphere
vol.
7
,
(
S1
)
9
-
9
.
(
2023
)
.
5609968 REAL-WORLD PATIENT-REPORTED OUTCOMES AND WEARABLE METRIC CORRELATIONS: REMOTE DAILY MONITORING FOR EXPLORATION OF QUALITY OF LIFE IN SICKLE CELL DISEASE
.
HemaSphere
vol.
7
,
(
S1
)
8
-
9
.
(
2023
)
.
5610107 LIVE OUTCOMES AND BIOMETRIC MONITORING: POTENTIAL IMPLICATIONS FOR PATIENT QUALITY OF LIFE IN SICKLE CELL DISEASE
.
HemaSphere
vol.
7
,
(
S1
)
54
-
54
.
(
2023
)
.
5613294 BASELINE PATIENT CHARACTERISTICS FROM A UK EARLY ACCESS TO MEDICINES SCHEME (EAMS) WITH VOXELOTOR, A HBS POLYMERIZATION INHIBITOR, FOR THE TREATMENT OF HEMOLYTIC ANEMIA DUE TO SICKLE CELL DISEASE
.
HemaSphere
vol.
7
,
(
S1
)
16
-
17
.
(
2023
)
.
5613396 DIFFERENCE IN HOSPITALIZATIONS AND ANNUAL BED DAYS FOR PATIENTS WITH SICKLE CELL DISEASE BY VARYING LEVELS OF ANEMIA: A RETROSPECTIVE ANALYSIS OF LINKED PRIMARY AND SECONDARY CARE DATABASES IN ENGLAND
.
HemaSphere
vol.
7
,
(
S1
)
32
-
33
.
(
2023
)
.
Incidence rates for acute complications in HbSS East London newborn sickle cell cohort (ELNSCS)
.
BRITISH JOURNAL OF HAEMATOLOGY
.
vol.
201
,
40
-
40
.
(
2023
)
.
Treatment of sickle cell disease with Voxelotor through the UK early access to medicines scheme
.
BRITISH JOURNAL OF HAEMATOLOGY
.
vol.
201
,
5
-
6
.
(
2022
)
.
Association entre le taux d’hémoglobine et les atteintes d’organes cibles dans la drépanocytose : analyse rétrospective d’une base de données de soins primaires et secondaires en Angleterre
.
La Revue de Médecine Interne
vol.
43
,
A441
-
A441
.
(
2022
)
.
ASSOCIATION BETWEEN HEMOGLOBIN LEVELS AND END-ORGAN DAMAGE IN SICKLE CELL DISEASE: A RETROSPECTIVE LINKED PRIMARY AND SECONDARY CARE DATABASE ANALYSIS IN ENGLAND
.
Hematology, Transfusion and Cell Therapy
vol.
44
,
S10
-
S11
.
(
2022
)
.
P-037: EXPLORATION OF THE REAL-WORLD IMPACTS OF SLEEP UPON QUALITY OF LIFE IN PATIENTS WITH SICKLE CELL DISEASE
.
HemaSphere
vol.
6
,
35
-
35
.
(
2022
)
.
P1488: ASSOCIATION BETWEEN HEMOGLOBIN LEVELS AND END-ORGAN DAMAGE IN SICKLE CELL DISEASE: A RETROSPECTIVE LINKED PRIMARY AND SECONDARY CARE DATABASE ANALYSIS IN ENGLAND
.
HemaSphere
vol.
6
,
1370
-
1371
.
(
2022
)
.
P1493: CHRONIC COMPLICATIONS IN THE EAST LONDON SICKLE NEWBORN COHORT STUDY (ELSNCS)
.
HemaSphere
vol.
6
,
1375
-
1376
.
(
2022
)
.
Sécurité et efficacité à long terme du voxelotor chez des patients atteints de drépanocytose : résultats d’une étude d’extension en ouvert de l’essai de phase 3 HOPE
.
La Revue de Médecine Interne
vol.
43
,
A202
-
A202
.
(
2022
)
.
A retrospective comparative study of the effect of automated exchange transfusion <i>versus</i> other treatment modalities on vaso-occlusive crisis admission rates in patients with sickle cell disease
.
BRITISH JOURNAL OF HAEMATOLOGY
.
vol.
197
,
138
-
139
.
(
2022
)
.
Challenges of acute and chronic pain management in sickle cell disease: Outcomes of an English national audit
.
BRITISH JOURNAL OF HAEMATOLOGY
.
vol.
197
,
11
-
12
.
(
2022
)
.
P131: REAL-TIME VACCINATION IMPACTS IN SICKLE CELL DISEASE: A REAL-WORLD PATIENT CASE STUDY FOR INFLUENZA AND COVID-19 VACCINATION
.
HemaSphere
vol.
6
,
32
-
33
.
(
2022
)
.
S110: ACUTE AND CHRONIC PAIN MANAGEMENT IN SICKLE CELL DISEASE: OUTCOMES OF AN ENGLISH NATIONAL AUDIT
.
HemaSphere
vol.
6
,
6
-
6
.
(
2022
)
.
S118: 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
.
HemaSphere
vol.
6
,
9
-
10
.
(
2021
)
.
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
.
Blood
vol.
138
,
(
Supplement 1
)
3114
-
3114
.
(
2021
)
.
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
.
Blood
vol.
138
,
(
Supplement 1
)
3093
-
3093
.
(
2021
)
.
Routine management, healthcare resource use and patient and carer‐reported outcomes of patients with transfusion‐dependent β‐thalassaemia in the United Kingdom: A mixed methods observational study
.
eJHaem
vol.
2
,
(
4
)
738
-
749
.
(
2021
)
.
A non-injected opioid analgesia protocol for acute pain crisis in adolescents and adults with sickle cell disease
.
British Journal of Pain
vol.
16
,
(
2
)
179
-
190
.
(
2021
)
.
The parental perspective of thalassaemia in Bangladesh: lack of knowledge, regret, and barriers
.
Orphanet Journal of Rare Diseases
vol.
16
,
(
1
)
Article
315
,
(
2021
)
.
The Parental Perspective of Thalassaemia in Bangladesh: Lack of knowledge, Regret, and Barriers
.
(
2021
)
.
Salvage of refractory post‐transfusion hyperhaemolysis by targeting hyperinflammation and macrophage activation with tocilizumab
.
Transfusion Medicine
vol.
32
,
(
5
)
437
-
440
.
(
2020
)
.
The role of haematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene editing
.
The Lancet Haematology
vol.
7
,
(
12
)
e902
-
e911
.
(
2020
)
.
Higher Hemoglobin Levels Achieved with Voxelotor Are Associated with Lower Vaso-occlusive Crisis Incidence: 72-Week Analysis from the HOPE Study
.
Blood
vol.
136
,
(
Supplement 1
)
31
-
32
.
(
2020
)
.
Improvement in the Clinical Global Impression of Change with Voxelotor in Patients with Sickle Cell Disease in the Phase 3 HOPE Trial
.
Blood
vol.
136
,
(
Supplement 1
)
5
-
6
.
(
2020
)
.
Co‐morbidities and mortality associated with transfusion‐dependent beta‐thalassaemia in patients in England: a 10‐year retrospective cohort analysis
.
British Journal of Haematology
vol.
191
,
(
5
)
897
-
905
.
(
2020
)
.
Real-time national survey of COVID-19 in hemoglobinopathy and rare inherited anemia patients
.
Haematologica
vol.
105
,
(
11
)
2651
-
2654
.
(
2020
)
.
Effect of HBB genotype on survival in a cohort of transfusion-dependent thalassemia patients in Cyprus
.
Haematologica
vol.
106
,
(
9
)
2458
-
2468
.
(
2020
)
.
Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial
.
The Lancet Haematology
vol.
7
,
(
6
)
e469
-
e478
.
(
2020
)
.
Protecting vulnerable patients with inherited anaemias from unnecessary death during the COVID‐19 pandemic
.
British Journal of Haematology
vol.
189
,
(
4
)
635
-
639
.
(
2020
)
.
Serial prophylactic exchange blood transfusion in pregnant women with sickle cell disease (TAPS-2): study protocol for a randomised controlled feasibility trial
.
Trials
vol.
21
,
(
1
)
Article
347
,
(
2020
)
.
American Society of Hematology 2020 guidelines for sickle cell disease: prevention, diagnosis, and treatment of cerebrovascular disease in children and adults
.
Blood Advances
vol.
4
,
(
8
)
1554
-
1588
.
(
2020
)
.
Lack of knowledge and misperceptions about thalassaemia among college students in Bangladesh: a cross-sectional baseline study
.
Orphanet Journal of Rare Diseases
vol.
15
,
(
1
)
Article
54
,
(
2020
)
.
Hydroxyurea therapy does not impact TCD velocity in the East London sickle cell newborn cohort study
.
BRITISH JOURNAL OF HAEMATOLOGY
.
vol.
189
,
146
-
147
.
(
2020
)
.
Rh antibodies in patients with sickle cell disease - an on-going challenge
.
BRITISH JOURNAL OF HAEMATOLOGY
.
vol.
189
,
166
-
166
.
(
2019
)
.
Not being heard: barriers to high quality unplanned hospital care during young people’s transition to adult services – evidence from ‘this sickle cell life’ research
.
BMC Health Services Research
vol.
19
,
(
1
)
Article
876
,
(
2019
)
.
Incidence of Vaso-Occlusive Crisis Does Not Increase with Achieving Higher Hemoglobin Levels on Voxelotor Treatment or after Discontinuation: Analyses of the HOPE Study
.
Blood
vol.
134
,
(
Supplement_1
)
2313
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2313
.
(
2019
)
.
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
.
Blood
vol.
134
,
(
Supplement_1
)
3550
-
3550
.
(
2019
)
.
PRO146 HEALTH-RELATED QUALITY OF LIFE AND PRODUCTIVITY/ACTIVITY IMPAIRMENT IN PATIENTS WITH TRANSFUSION DEPENDENT B-THALASSAEMIA IN THE UK NHS: DATA FROM A UK MULTICENTRE OBSERVATIONAL STUDY
.
Value in Health
vol.
22
,
S868
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S869
.
(
2019
)
.
Evaluation of a Non-Parenteral Opioid Analgesia Protocol for Acute Sickle Cell Pain Episodes in Children
.
Journal of Clinical Medicine
vol.
8
,
(
10
)
1728
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1728
.
(
2019
)
.
The Role of Family Functioning in the Development of Executive Functions in Preschool Children with Sickle Cell Anemia
.
Developmental Neuropsychology
vol.
44
,
(
6
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452
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467
.
(
2019
)
.
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease
.
New England Journal of Medicine
vol.
381
,
(
6
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509
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519
.
(
2019
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Management of sickle cell disease: management of acute episodes in the community and in hospital
.
Paediatrics and Child Health
vol.
29
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8
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345
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351
.
(
2019
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Management of sickle cell disease: outpatient and community aspects
.
Paediatrics and Child Health
vol.
29
,
(
8
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352
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358
.
(
2019
)
.
PF785 CHANGING CAUSES OF MORTALITY IN TDT DURING THE ERA OF ORAL CHELATION THERAPY FROM 2000 TO 2018
.
HemaSphere
vol.
3
,
(
S1
)
346
-
347
.
(
2019
)
.
S144 A MULTICENTRE, RANDOMIZED, NON‐INFERIORITY TRIAL COMPARING THE EFFICACY OF DEFERIPRONE VERSUS DEFERASIROX IN PEDIATRIC PATIENTS AFFECTED BY TRANSFUSION‐DEPENDENT HEMOGLOBINOPATHIES (DEEP‐2 TRIAL)
.
HemaSphere
vol.
3
,
(
S1
)
23
-
24
.
(
2019
)
.
S147 RESULTS FROM THE RANDOMIZED PLACEBO‐CONTROLLED PHASE 3 HOPE TRIAL OF VOXELOTOR IN ADULTS AND ADOLESCENTS WITH SICKLE CELL DISEASE
.
HemaSphere
vol.
3
,
(
S1
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25
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26
.
(
2019
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.
Development and validation of the Satisfaction with Treatment for Pain Questionnaire (STPQ) among patients with sickle cell disease
.
British Journal of Haematology
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vol.
187
,
105
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116
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2019
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Psychometric analysis of the adult sickle cell quality of life measurement information system (ACSQ-Me) in a UK population
.
Health and Quality of Life Outcomes
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17
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1
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Article
74
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2019
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A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease
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Blood
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17
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1865
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1875
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2019
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FRI-437-Diagnosis of sickle cell liver disease may be aided by non-invasive tests
.
Journal of Hepatology
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vol.
70
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e586
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e587
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(
2019
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Extreme cardiac iron loading in transfusion-dependent thalassaemia major: cardiac T2* and T1 mapping guiding treatment
.
European Heart Journal
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40
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(
43
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3578
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3578
.
(
2019
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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
.
BRITISH JOURNAL OF HAEMATOLOGY
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185
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113
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114
.
(
2019
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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
.
BRITISH JOURNAL OF HAEMATOLOGY
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185
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114
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115
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2019
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Causes, management and outcomes of extreme paediatric thrombocytosis
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BRITISH JOURNAL OF HAEMATOLOGY
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185
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108
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108
.
(
2019
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HEALTH-RELATED QUALITY OF LIFE AND PRODUCTIVITY/ACTIVITY IMPAIRMENT IN PATIENTS WITH TRANSFUSION DEPENDENT B-THALASSAEMIA IN THE UK NHS: DATA FROM A UK MULTICENTRE OBSERVATIONAL STUDY
.
VALUE IN HEALTH
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S868
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S869
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Patients lost to follow-up in the East London Newborn Sickle Cell Cohort Study
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BRITISH JOURNAL OF HAEMATOLOGY
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185
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123
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124
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2019
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Peer to peer mentoring for patients with Sickle Cell Disease - interim analysis of results from a pilot programme in East London
.
BRITISH JOURNAL OF HAEMATOLOGY
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185
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116
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116
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(
2018
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Temperament in preschool children with sickle cell anaemia
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Archives of Disease in Childhood
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105
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9
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900
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902
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(
2018
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Hydroxyurea Therapy Does Not Impact Current Survival Estimates in the East London Sickle Cell Newborn Cohort Study
.
Blood
vol.
132
,
(
Supplement 1
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3655
-
3655
.
(
2018
)
.
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
.
vol.
132
,
505
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505
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(
2018
)
.
Developing a risk-based composite neurologic outcome for a trial of hydroxyurea in young children with sickle cell disease
.
Clinical Trials
vol.
16
,
(
1
)
20
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31
.
(
2018
)
.
Newborn screening for sickle cell disease in Europe: recommendations from a Pan‐European Consensus Conference
.
British Journal of Haematology
vol.
183
,
(
4
)
648
-
660
.
(
2018
)
.
Chronic Hepatitis C Virus Infection in Haemophilic Patients: Clinical Significance of Viral Genotype
.
Thrombosis and Haemostasis
vol.
74
,
(
05
)
1259
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1264
.
(
2018
)
.
Executive performance on the preschool executive task assessment in children with sickle cell anemia and matched controls
.
Child Neuropsychology
vol.
25
,
(
2
)
278
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285
.
(
2018
)
.
Assessment of Executive Functions in Preschool Children With Sickle Cell Anemia
.
Journal of the International Neuropsychological Society
vol.
24
,
(
9
)
949
-
954
.
(
2018
)
.
Impact of voxelotor (GBT440) on unconjugated bilirubin and jaundice in sickle cell disease
.
Hematology Reports
vol.
10
,
(
2
)
(
2018
)
.
A Case Report of Dehydrated Hereditary Stomatocytosis and Spherocytosis
.
BRITISH JOURNAL OF HAEMATOLOGY
.
vol.
181
,
125
-
125
.
(
2018
)
.
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