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.
How I treat cytopenias after CAR T-cell therapy.
Blood
; 141(20): 2460-2469, 2023 05 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-36800563
3.
Hypomethylating agents are associated with high rates of hematologic toxicity in patients with secondary myeloid neoplasms developing after acquired aplastic anemia.
Haematologica
; 2024 Apr 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-38634143
4.
Genomic profiling of pediatric hematologic malignancies and diagnosis of cancer predisposition syndromes: tumor-only versus paired tumor-normal sequencing.
Haematologica
; 2024 Feb 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-38385299
5.
Inherited bone marrow failure with macrothrombocytopenia due to germline tubulin beta class I (TUBB) variant.
Br J Haematol
; 200(2): 222-228, 2023 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36207145
6.
B-cell acute lymphoblastic leukemia and juvenile xanthogranuloma in a patient with ETV6 thrombocytopenia and leukemia predisposition syndrome: novel clinical presentation and perspective.
Haematologica
; 2023 Nov 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-38031764
7.
Comparison of hematopoietic cell transplant conditioning regimens for hemophagocytic lymphohistiocytosis disorders.
J Allergy Clin Immunol
; 149(3): 1097-1104.e2, 2022 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-34375618
8.
Hematologic abnormalities in Aicardi Goutières Syndrome.
Mol Genet Metab
; 136(4): 324-329, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35786528
9.
Guideline for Children With Cancer Receiving General Anesthesia for Procedures and Imaging.
J Pediatr Hematol Oncol
; 44(4): e859-e865, 2022 05 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-35235547
10.
SLC25A38 congenital sideroblastic anemia: Phenotypes and genotypes of 31 individuals from 24 families, including 11 novel mutations, and a review of the literature.
Hum Mutat
; 42(11): 1367-1383, 2021 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-34298585
11.
Model system identification of novel congenital heart disease gene candidates: focus on RPL13.
Hum Mol Genet
; 28(23): 3954-3969, 2019 12 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31625562
12.
Diagnostic work-up for severe aplastic anemia in children: Consensus of the North American Pediatric Aplastic Anemia Consortium.
Am J Hematol
; 96(11): 1491-1504, 2021 11 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34342889
13.
TFEB Overexpression, Not mTOR Inhibition, Ameliorates RagCS75Y Cardiomyopathy.
Int J Mol Sci
; 22(11)2021 May 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-34071043
14.
The Natural History of BK Polyomavirus and the Host Immune Response After Stem Cell Transplantation.
Clin Infect Dis
; 71(12): 3044-3054, 2020 12 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-31851312
15.
Partially CD3+-Depleted Unrelated and Haploidentical Donor Peripheral Stem Cell Transplantation Has Favorable Graft-versus-Host Disease and Survival Rates in Pediatric Hematologic Malignancy.
Biol Blood Marrow Transplant
; 26(3): 493-501, 2020 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-31765697
16.
A study assessing the feasibility of randomization of pediatric and young adult patients between matched unrelated donor bone marrow transplantation and immune-suppressive therapy for newly diagnosed severe aplastic anemia: A joint pilot trial of the North American Pediatric Aplastic Anemia Consortium and the Pediatric Transplantation and Cellular Therapy Consortium.
Pediatr Blood Cancer
; 67(10): e28444, 2020 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-32776425
17.
Diagnosis and treatment of pediatric myelodysplastic syndromes: A survey of the North American Pediatric Aplastic Anemia Consortium.
Pediatr Blood Cancer
; 67(10): e28652, 2020 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-32779892
18.
Recessive TAF1A mutations reveal ribosomopathy in siblings with end-stage pediatric dilated cardiomyopathy.
Hum Mol Genet
; 26(15): 2874-2881, 2017 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28472305
19.
CD3+/CD19+ Depleted Matched and Mismatched Unrelated Donor Hematopoietic Stem Cell Transplant with Targeted T Cell Addback Is Associated with Excellent Outcomes in Pediatric Patients with Nonmalignant Hematologic Disorders.
Biol Blood Marrow Transplant
; 25(3): 549-555, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30312755
20.
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