Detalhe da pesquisa
1.
Novel therapeutics and future directions for refractory immune thrombocytopenia.
Br J Haematol
; 203(1): 65-78, 2023 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-37735554
2.
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent ß-Thalassemia.
N Engl J Med
; 382(13): 1219-1231, 2020 03 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-32212518
3.
The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective.
Blood
; 138(18): 1677-1690, 2021 11 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33895800
4.
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
5.
Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A-Final efficacy and safety results from the NuProtect study.
Eur J Haematol
; 111(4): 544-552, 2023 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-37439123
6.
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
7.
Which pediatric ITP patients will go on to lupus?
Blood
; 143(16): 1553-1554, 2024 Apr 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-38635255
8.
Limited sampling strategies for accurate determination of extended half-life factor VIII pharmacokinetics in severe haemophilia A patients.
Haemophilia
; 27(3): 408-416, 2021 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-33742733
9.
Comorbidities and complications in adults with pyruvate kinase deficiency.
Eur J Haematol
; 106(4): 484-492, 2021 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-33370479
10.
Pyruvate kinase deficiency in children.
Pediatr Blood Cancer
; 68(9): e29148, 2021 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-34125488
11.
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
12.
Regional variation and cost implications of prescribed extended half-life factor concentrates among U.S. Haemophilia Treatment Centres for patients with moderate and severe haemophilia.
Haemophilia
; 25(4): 668-675, 2019 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-30993845
13.
Second-line treatments in children with immune thrombocytopenia: Effect on platelet count and patient-centered outcomes.
Am J Hematol
; 94(7): 741-750, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30945320
14.
Physician decision making in selection of second-line treatments in immune thrombocytopenia in children.
Am J Hematol
; 93(7): 882-888, 2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29659042
15.
Platelet function tests, independent of platelet count, are associated with bleeding severity in ITP.
Blood
; 126(7): 873-9, 2015 Aug 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-26138687
16.
Safety and pharmacokinetics of the oral iron chelator SP-420 in ß-thalassemia.
Am J Hematol
; 92(12): 1356-1361, 2017 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-28940308
17.
Increasing observation rates in low-risk pediatric immune thrombocytopenia using a standardized clinical assessment and management plan (SCAMP® ).
Pediatr Blood Cancer
; 64(5)2017 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-27781392
18.
Ringed sideroblasts in ß-thalassemia.
Pediatr Blood Cancer
; 64(5)2017 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-27808451
19.
Safety and efficacy of recombinant factor VIIa by pediatric age cohort: reassessment of compassionate use and trial data supporting US label.
Pediatr Blood Cancer
; 63(10): 1822-8, 2016 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-27232114
20.
Center-Based Quality Initiative Targets Youth Preparedness for Medical Independence: HEMO-Milestones Tool in a Comprehensive Hemophilia Clinic Setting.
Pediatr Blood Cancer
; 63(3): 499-503, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-26496140