Thompson, Alexis A.

Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia

Manco-Johnson, M. J., Abshire, T. C., Shapiro, A. D., Riske, B., Hacker, M. R., Kilcoyne, R., Ingram, J. D., Manco-Johnson, M. L., Funk, S., Jacobson, L., et al. (2007) · New England Journal of Medicine

Hemophilia Treatment and ResearchPlatelet Disorders and Treatments

Cited by 1,950

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., Tai, S. J., Ragni, M. V., Thompson, A., Ozelo, M., et al. (2003) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 749

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E. J., Ribeil, J.-A., Hongeng, S., Magrin, E., Schiller, G. J., Payen, E., Semeraro, M., et al. (2018) · New England Journal of Medicine

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 684

Controlled Trial of Transfusions for Silent Cerebral Infarcts in Sickle Cell Anemia

DeBaun, M. R., Gordon, M., McKinstry, R. C., Noetzel, M. J., White, D. A., Sarnaik, S. A., Meier, E. R., Howard, T. H., Majumdar, S., Inusa, B. P. D., et al. (2014) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersBlood transfusion and management

Cited by 545

Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial

Ware, R. E., Davis, B. R., Schultz, W. H., Brown, R. C., Aygun, B., Sarnaik, S., Odame, I., Fuh, B., George, A., Owen, W., et al. (2016) · The Lancet

Hemoglobinopathies and Related DisordersNeonatal Health and Biochemistry

Cited by 515

A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia.

Cappellini, M. D., Viprakasit, V., Taher, A. T., Georgiev, P., Kuo, K. H. M., Coates, T., Voskaridou, E., Liew, H.-K., Pazgal-Kobrowski, I., Forni, G. L., et al. (2020) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersBlood transfusion and management

Cited by 332

Mutations in TRNT1 cause congenital sideroblastic anemia with immunodeficiency, fevers, and developmental delay (SIFD)

Chakraborty, P. K., Schmitz-Abe, K., Kennedy, E. K., Mamady, H., Naas, T., Durie, D., Campagna, D. R., Lau, A., Sendamarai, A. K., Wiseman, D. H., et al. (2014) · Blood

Iron Metabolism and DisordersRNA and protein synthesis mechanisms

Cited by 190

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

Hsieh, M. M., Bonner, M., Pierciey, F. J., Uchida, N., Rottman, J., Demopoulos, L., Schmidt, M., Kanter, J., Walters, M. C., Thompson, A. A., et al. (2020) · Blood Advances

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 152

Effects of hydroxyurea treatment for patients with hemoglobin SC disease

Luchtman‐Jones, L., Pressel, S., Hilliard, L., Brown, R. C., Smith, M. G., Thompson, A. A., Lee, M. T., Rothman, J., Rogers, Z. R., Owen, W., et al. (2016) · American Journal of Hematology

Hemoglobinopathies and Related DisordersHemophilia Treatment and Research

Cited by 86

Guidelines for the Standard Monitoring of Patients With Thalassemia: Report of the Thalassemia Longitudinal Cohort

Tubman, V. N., Fung, E. B., Vogiatzi, M., Thompson, A. A., Rogers, Z. R., Neufeld, E. J., & Kwiatkowski, J. L. (2015) · Journal of Pediatric Hematology/Oncology

Hemoglobinopathies and Related DisordersBlood transfusion and management

Cited by 68

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Kanter, J., Tisdale, J. F., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Huang, W., Ribeil, J.-A., et al. (2019) · Blood

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 21

Efficacy, Safety, and Health-Related Quality of Life (HRQOL) in Patients with Sickle Cell Disease (SCD) Who Have Received Lovotibeglogene Autotemcel (Lovo-cel) Gene Therapy: Up to 60 Months of Follow-up

Kanter, J., Thompson, A. A., Kwiatkowski, J. L., Parikh, S., Mapara, M., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Gupta, A. O., Zhang, L., et al. (2023) · Blood

Hemoglobinopathies and Related DisordersNeurogenetic and Muscular Disorders Research

Cited by 15

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Thompson, A. A., Olson, T. S., Walters, M. C., Porter, J. B., Schneiderman, J., Hongeng, S., Kulozik, A., Cavazzana, M., Sauer, M. G., Thrasher, A. J., et al. (2023) · Blood

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 6