Detalhe da pesquisa
1.
Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or ß-thalassaemia: an open-label, multicentre, phase 2 study.
Lancet
; 400(10351): 493-501, 2022 08 13.
Artigo
Inglês
| MEDLINE | ID: mdl-35964609
2.
Development of a Thalassemia International Prognostic Scoring System (TIPSS).
Blood Cells Mol Dis
; 99: 102710, 2023 03.
Artigo
Inglês
| MEDLINE | ID: mdl-36463683
3.
COVID-19 Infection and Outcomes in Newborn Screening Cohorts of Sickle Cell Trait and Sickle Cell Disease in Michigan and Georgia.
J Pediatr Hematol Oncol
; 45(4): 174-180, 2023 05 01.
Artigo
Inglês
| MEDLINE | ID: mdl-37083273
4.
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
Inglês
| MEDLINE | ID: mdl-34775608
5.
Primary HBB gene mutation severity and long-term outcomes in a global cohort of ß-thalassaemia.
Br J Haematol
; 196(2): 414-423, 2022 01.
Artigo
Inglês
| MEDLINE | ID: mdl-34697800
6.
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.
N Engl J Med
; 381(6): 509-519, 2019 08 08.
Artigo
Inglês
| MEDLINE | ID: mdl-31199090
7.
Transitioning Adolescents With Sickle Cell Disease From Pediatric to Adult Care: Results From a New Survey of Health Care Professionals.
J Pediatr Hematol Oncol
; 44(7): e999-e1005, 2022 10 01.
Artigo
Inglês
| MEDLINE | ID: mdl-35700397
8.
Time to rethink haemoglobin threshold guidelines in sickle cell disease.
Br J Haematol
; 195(4): 518-522, 2021 11.
Artigo
Inglês
| MEDLINE | ID: mdl-34131897
9.
A complication risk score to evaluate clinical severity of thalassaemia syndromes.
Br J Haematol
; 192(3): 626-633, 2021 02.
Artigo
Inglês
| MEDLINE | ID: mdl-33216983
10.
A Phase 3 Trial of l-Glutamine in Sickle Cell Disease.
N Engl J Med
; 379(3): 226-235, 2018 Jul 19.
Artigo
Inglês
| MEDLINE | ID: mdl-30021096
11.
Gene Therapy in Patients with Transfusion-Dependent ß-Thalassemia.
N Engl J Med
; 378(16): 1479-1493, 2018 04 19.
Artigo
Inglês
| MEDLINE | ID: mdl-29669226
12.
Pituitary iron and factors predictive of fertility status in transfusion dependent thalassemia.
Haematologica
; 106(6): 1740-1744, 2021 06 01.
Artigo
Inglês
| MEDLINE | ID: mdl-33121232
13.
Improving healthspan among people with sickle cell disease: Leveraging precision health in an era of treatments with curative intent.
Am J Hematol
; 2024 May 16.
Artigo
Inglês
| MEDLINE | ID: mdl-38752374
14.
Evaluation of Mandible Fractures in Patients With Sickle Cell Anemia-A Nationwide Study.
J Oral Maxillofac Surg
; 77(7): 1418-1422, 2019 Jul.
Artigo
Inglês
| MEDLINE | ID: mdl-30707980
15.
Variability of homozygous sickle cell disease: The role of alpha and beta globin chain variation and other factors.
Blood Cells Mol Dis
; 70: 66-77, 2018 05.
Artigo
Inglês
| MEDLINE | ID: mdl-28689691
16.
Transfusion practices and complications in thalassemia.
Transfusion
; 58(12): 2826-2835, 2018 12.
Artigo
Inglês
| MEDLINE | ID: mdl-30260477
17.
Epidemiologic and clinical characteristics of nontransfusion-dependent thalassemia in the United States.
Pediatr Blood Cancer
; 65(7): e27067, 2018 07.
Artigo
Inglês
| MEDLINE | ID: mdl-29637688
18.
Simvastatin reduces vaso-occlusive pain in sickle cell anaemia: a pilot efficacy trial.
Br J Haematol
; 177(4): 620-629, 2017 05.
Artigo
Inglês
| MEDLINE | ID: mdl-28369718
19.
Risk of mortality from anemia and iron overload in nontransfusion-dependent ß-thalassemia.
Am J Hematol
; 97(2): E78-E80, 2022 02 01.
Artigo
Inglês
| MEDLINE | ID: mdl-34862982
20.
Long-term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: Results from a 5-year observational study (ENTRUST).
Pediatr Blood Cancer
; 64(9)2017 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-28296163