Detalhe da pesquisa
1.
Betibeglogene Autotemcel Gene Therapy for Non-ß0/ß0 Genotype ß-Thalassemia.
N Engl J Med
; 386(5): 415-427, 2022 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34891223
2.
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.
N Engl J Med
; 386(7): 617-628, 2022 02 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-34898139
3.
Real-world evidence: Long-term safety of deferiprone in a large cohort of patients with sickle cell disease enrolled in a registry for up to 10 years.
Am J Hematol
; 2024 Mar 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38429922
4.
Safety of Ultrasound Contrast Agents in Pediatric Patients With Sickle Cell Disease and Trait.
J Ultrasound Med
; 43(1): 189-200, 2024 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-37929626
5.
Health-related quality of life in patients with ß-thalassemia: Data from the phase 3 BELIEVE trial of luspatercept.
Eur J Haematol
; 111(1): 113-124, 2023 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-37095595
6.
Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.
Am J Hematol
; 98(1): 11-22, 2023 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36161320
7.
Return visit rates after an emergency department discharge for children with sickle cell pain episodes.
Pediatr Blood Cancer
; 70(10): e30553, 2023 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-37458568
8.
An update on the US adult thalassaemia population: a report from the CDC thalassaemia treatment centres.
Br J Haematol
; 196(2): 380-389, 2022 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34775608
9.
Time to rethink haemoglobin threshold guidelines in sickle cell disease.
Br J Haematol
; 195(4): 518-522, 2021 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-34131897
10.
The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency.
Br J Haematol
; 192(6): 1092-1096, 2021 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-32463523
11.
Gene Therapy in Patients with Transfusion-Dependent ß-Thalassemia.
N Engl J Med
; 378(16): 1479-1493, 2018 04 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-29669226
12.
Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial.
JAMA
; 325(15): 1513-1523, 2021 04 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-33877274
13.
Summit on Exercise Collapse Associated with Sickle Cell Trait: Finding the "Way Ahead".
Curr Sports Med Rep
; 20(1): 47-56, 2021 Jan 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-33395130
14.
Clinical spectrum of pyruvate kinase deficiency: data from the Pyruvate Kinase Deficiency Natural History Study.
Blood
; 131(20): 2183-2192, 2018 05 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-29549173
15.
A patient-centered medical home model for comprehensive sickle cell care in infants and young children.
Pediatr Blood Cancer
; 67(6): e28275, 2020 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-32277797
16.
Association among sickle cell trait, fitness, and cardiovascular risk factors in CARDIA.
Blood
; 129(6): 723-728, 2017 02 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27856464
17.
Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.
Haematologica
; 104(10): 1974-1983, 2019 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-30948484
18.
Combination Oral Chelation in Adult Patients With Transfusion-dependent Thalassemia and High Iron Burden.
J Pediatr Hematol Oncol
; 41(1): e47-e50, 2019 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-30080754
19.
Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.
Biol Blood Marrow Transplant
; 24(6): 1216-1222, 2018 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-29374585
20.
Epidemiologic and clinical characteristics of nontransfusion-dependent thalassemia in the United States.
Pediatr Blood Cancer
; 65(7): e27067, 2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29637688