Genetic and Clinical Aspects of Hemoglobin Disorders

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Shah, N. C., Ngwube, A., Suresh, T., Sowa, A., Abraham, A., Anderson, E., Andreansky, M., Bhatia, M., Chaudhury, S., Cuvelier, G. D. E., Dalal, J., Grimley, M., Jacobsohn, D., Kamani, N., Krajewski, J., Krishnamurti, L., Saini, S., Skiles, J., & Shenoy, S. (2025). Impact of Abatacept Inclusive Graft Versus Host Disease Prophylaxis in Pediatric Stem Cell Transplantation for Hemoglobinopathy. Transplantation and Cellular Therapy. https://doi.org/10.1016/j.jtct.2025.06.015
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Wufsus, A., Delicou, S., El Rassi, F., Andemariam, B., Abboud, M. R., Kanter, J., Telen, M. J., Githanga, J., Inati, A., Idris, I. M., Navani, S., Wu, E., & Eisenberger, A. (2025). Etavopivat Reduces Incidence of Vaso-occlusive Crises in Patients With Sickle Cell Disease: Hibiscus Trial Phase 2 Results through 52 Weeks. Journal of Sickle Cell Disease, 2(Supplement_1). https://doi.org/10.1093/jscdis/yoaf013.002
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Mishkin, A. D. (2025). Screening Adolescents for Depression in Sickle Cell Disease: It All Starts with Getting the Patients Into Your Office. Pediatric Blood & Cancer. Portico. https://doi.org/10.1002/pbc.31688
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Naggayi, S. K., Kalibbala, D., Mboizi, V., Ssenkusu, J. M., Jin, Z., Rosano, C., Munube, D., Wambaka, B., Namazzi, R., Kasirye, P. G., Kabatabaazi, M., Nambatya, G., Murungi, S., Nabaggala, C., Nakafeero, M. R., Troidl, I. R., Opoka, R. O., Idro, R., Bangirana, P., & Green, N. S. (2025). Neurocognitive Gains Among Ugandan Children with Sickle Cell Anemia on Hydroxyurea: 18-month trial interim results. Blood Advances. https://doi.org/10.1182/bloodadvances.2024015073
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Mboizi, V., Nabaggala, C., Munube, D., Ssenkusu, J. M., Kasirye, P., Kamya, S., Kawooya, M. G., Boehme, A., Minja, F., Mupere, E., Opoka, R., Rosano, C., Idro, R., & Green, N. S. (2024). Hydroxyurea therapy for neurological and cognitive protection in pediatric sickle cell anemia in Uganda (BRAIN SAFE II): Protocol for a single-arm open label trial. Contemporary Clinical Trials Communications, 42, 101404. https://doi.org/10.1016/j.conctc.2024.101404
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Reisz, J. A., Earley, E. J., Nemkov, T., Key, A., Stephenson, D., Keele, G. R., Dzieciatkowska, M., Spitalnik, S. L., Hod, E. A., Kleinman, S., Roubinian, N. H., Gladwin, M. T., Hansen, K. C., Norris, P. J., Busch, M. P., Zimring, J. C., Churchill, G. A., Page, G. P., & D’Alessandro, A. (2024). Arginine metabolism is a biomarker of red blood cell and human aging. Aging Cell. Portico. https://doi.org/10.1111/acel.14388
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Akpan, I. J., Bogyo, K., Leeman-Neill, R. J., Wattacheril, J., & Francis, R. O. (2024). Congenital dyserythropoietic anemia type II and ineffective erythropoiesis: challenges in diagnosis and management. Frontiers in Hematology, 3. https://doi.org/10.3389/frhem.2024.1389820
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Manzoni, E., Carli, S., Gaignard, P., Schlieben, L. D., Hirano, M., Ronchi, D., Gonzales, E., Shimura, M., Murayama, K., Okazaki, Y., Barić, I., Petkovic Ramadza, D., Karall, D., Mayr, J., Martinelli, D., La Morgia, C., Primiano, G., Santer, R., Servidei, S., … Garone, C. (2024). Deoxyguanosine kinase deficiency: natural history and liver transplant outcome. Brain Communications, 6(3). https://doi.org/10.1093/braincomms/fcae160
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Bangirana, P., Boehme, A. K., Birabwa, A., Opoka, R. O., Munube, D., Mupere, E., Kasirye, P., Muwanguzi, G., Musiimenta, M., Ru, G., Green, N. S., & Idro, R. (2024). Neurocognitive impairment in Ugandan children with sickle cell anemia compared to sibling controls: a cross-sectional study. Frontiers in Stroke, 3. https://doi.org/10.3389/fstro.2024.1372949
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Verweij, E., Tura, A. K., Gure, T., Pyuza, J., Mchome, B., Kawaza, K., Moons, P., de Winter, D., van’t Oever, R., Spitalnik, S., van der Schoot, C. E., & van den Akker, T. (2024). Monoclonal RhD prophylaxis: high time to evaluate efficacy. The Lancet, 403(10429), 806–807. https://doi.org/10.1016/s0140-6736(23)01888-3
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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., Sheldon-Waniga, E., Gallagher, M., Gruppioni, K., Chawla, A., Elliot, H., Pierciey, F. J., Walters, M. C., & Tisdale, J. F. (2023). 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. Blood, 142(Supplement 1), 1051–1051. https://doi.org/10.1182/blood-2023-174229
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Proli, A., Sawas, A., Ho, G., Wang, J., & Fullerton, C. (2023). Methods for Identification of Blood Product Transfusions in a Cohort of Myelofibrosis Patients: An Assessment of Data Capture Using Insurance Claims and Electronic Health Record Abstraction. Blood, 142(Supplement 1), 7292–7292. https://doi.org/10.1182/blood-2023-189374
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Naggayi, S. K., Kalibala, D., Opoka, R., Rosano, C., Munube, D., Kasirye, P., Mupere, E., Mboizi, V., Ssenkusu, J., Green, N. S., Idro, R., & Bangirana, P. (2023). Neurocognitive Improvement with Hydroxyurea Therapy in Children with Sickle Cell Anemia in Uganda: Interim Analysis at Month 18. Blood, 142(Supplement 1), 275–275. https://doi.org/10.1182/blood-2023-190687
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Hanna, R., Frangoul, H., McKinney, C., Pineiro, L., Mapara, M., Dalal, J., Chang, K.-H., Jaskolka, M., Kim, K., Farrington, D. L., Wally, M., Mei, B., Lawal, A., Afonja, O. O., & Walters, M. C. (2023). AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia. Blood, 142(Supplement 1), 4996–4996. https://doi.org/10.1182/blood-2023-187397
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Kalibala, D., Nabaggala, C., Turyagyenda, L., Mboizi, V., Naggayi, S. K., Kabatabaazi, M., Rosano, C., Munube, D., Kasirye, P., Ssenkusu, J., Opoka, R., Bangirana, P., Green, N. S., Idro, R., & Mupere, E. (2023). Effect of Hydroxyurea Treatment on Body Composition in Children with Sickle Cell Anemia in Uganda Using Bioelectrical Impedance Analysis (BIA). Blood, 142(Supplement 1), 1136–1136. https://doi.org/10.1182/blood-2023-190181
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Namazzi, R., Idro, R., Kalibala, D., Ssenkusu, J., Akun, P., Mboizi, V., Gondwe, T. N., Munube, D., Kasirye, P., Nabaggala, C., John, C. C., Green, N. S., Robberstat, B., Hensbroek, B. van, Kuile, F. ter, Phiri, K. S., & Opoka, R. (2023). Malaria in Children with Sickle Cell Anaemia in Areas with Low, Moderate and High Transmission in Uganda. Blood, 142(Supplement 1), 5327–5327. https://doi.org/10.1182/blood-2023-189066
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Green, N. S., Manwani, D., Aygun, B., Appiah-Kubi, A., Smith-Whitley, K., Jia, H., & Smaldone, A. (2023). Habit Efficacy Trial: A Multi-Site Randomized Controlled Trial of Community Health Worker Support to Increase Hydroxyurea Adherence of Youth with Sickle Cell Disease. Blood, 142(Supplement 1), 3901–3901. https://doi.org/10.1182/blood-2023-190031
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Columbia Affiliation
Green, N. S., Rosano, C., Bangirana, P., Opoka, R., Munube, D., Kasirye, P., Kawooya, M., Lubowa, S. K., Mupere, E., Conroy, A., Minja, F. J., Boehme, A. K., Kang, M. S., Honig, L. S., & Idro, R. (2023). Neurofilament light chain: A potential biomarker for cerebrovascular disease in children with sickle cell anaemia. British Journal of Haematology, 203(3), 460–467. Portico. https://doi.org/10.1111/bjh.19036
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Carvalho Poyraz, F., Ghoshal, S., Agarwal, S., Park, S., Claassen, J., Hod, E., & Roh, D. (2023). P‐PB‐5 | Anemia Development During Intracerebral Hemorrhage Hospitalization is Associated with Poor Long‐Term Outcomes. Transfusion, 63(S5). Portico. https://doi.org/10.1111/trf.293_17554
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Coughlan, A., Klijn, S. L., Xiao, H., White, B., Litkiewicz, M., Kadambi, A., Sichevaya, A., Hnoosh, A., Miteva, D., & Yucel, A. (2023). POSTER: MDS-008 Impact of Timing of Achievement of Red Blood Cell-Transfusion Independence (RBC-TI) on Clinical Outcomes and Resource Utilization in Lower-Risk Myelodysplastic Syndromes (LR-MDS). Clinical Lymphoma Myeloma and Leukemia, 23, S180. https://doi.org/10.1016/s2152-2650(23)00575-x
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Hanna, R., Frangoul, H., Mckinney, C., Pineiro, L., Mapara, M., Chang, K.-H., Jaskolka, M., Kim, K., Rizk, M., Afonja, O., Lawal, A., & Walters, M. (2023). S264: EDIT-301 SHOWS PROMISING PRELIMINARY SAFETY AND EFFICACY RESULTS IN THE PHASE I/II CLINICAL TRIAL (RUBY) OF PATIENTS WITH SEVERE SICKLE CELL DISEASE USING HIGHLY SPECIFIC AND EFFICIENT ASCAS12A ENZYME. HemaSphere, 7(S3), e05170e0. https://doi.org/10.1097/01.hs9.0000967968.05170.e0
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Locatelli, F., Lang, P., Corbacioglu, S., LI, A., De La Fuente, J., Wall, D., Meisel, R., Shah, A. J., Liem, R., Mapara, M., Carpenter, B., Kwiatkowski, J., Domenica Cappellini, M., Kattamis, A., Sheth, S., Grupp, S., Kohli, P., Shi, D., Ross, L., … Frangoul, H. (2023). S270: TRANSFUSION INDEPENDENCE AFTER EXAGAMGLOGENE AUTOTEMCEL IN PATIENTS WITH TRANSFUSION-DEPENDENT ΒETA-THALASSEMIA. HemaSphere, 7(S3), e8473180. https://doi.org/10.1097/01.hs9.0000967992.84731.80
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Kudose, S., Cossey, L. N., Canetta, P. A., Sekulic, M., Vanbeek, C. A., Huls, F. B., Gupta, I., Bu, L., Alexander, M. P., Cornell, L. D., Fidler, M. E., Markowitz, G. S., Larsen, C. P., D’Agati, V. D., Nasr, S. H., & Santoriello, D. (2023). Clinicopathologic Spectrum of Lysozyme-Associated Nephropathy. Kidney International Reports, 8(8), 1585–1595. https://doi.org/10.1016/j.ekir.2023.05.007
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Reeves, S. L., Peng, H. K., Wing, J. J., Cogan, L. W., Goel, A., Anders, D., Green, N. S., Lisabeth, L. D., & Dombkowski, K. J. (2023). Changes in Hydroxyurea Use Among Youths Enrolled in Medicaid With Sickle Cell Anemia After 2014 Revision of Clinical Guidelines. JAMA Network Open, 6(3), e234584. https://doi.org/10.1001/jamanetworkopen.2023.4584
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Stenger, E., Xiang, Y., Wetzel, M., Gillespie, S., Chellapandian, D., Shah, R., Arnold, S. D., Bhatia, M., Chaudhury, S., Eckrich, M. J., Kanter, J., Kasow, K. A., Krajewski, J., Nickel, R. S., Ngwube, A. I., Olson, T. S., Rangarajan, H. G., Wobma, H., Guilcher, G. M. T., … Abraham, A. (2023). Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance. Transplantation and Cellular Therapy, 29(1), 47.e1-47.e10. https://doi.org/10.1016/j.jtct.2022.10.012
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Green, N. S., Zapfel, A., Nnodu, O. E., Franklin, P., Tubman, V. N., Chirande, L., Kiyaga, C., Chunda-Liyoka, C., Awuonda, B., Ohene-Frempong, K., Inusa, B. P. D., Ware, R. E., Odame, I., Ambrose, E. E., Dogara, L. G., Oron, A. P., Willett, C., Thompson, A. A., Berliner, N., … Novelli, E. M. (2022). The Consortium on Newborn Screening in Africa for sickle cell disease: study rationale and methodology. Blood Advances, 6(24), 6187–6197. https://doi.org/10.1182/bloodadvances.2022007698
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Meacham, L. R., Pecker, L. H., Gee, B., & Mishkin, A. (2022). Incorporating gonadal health counseling into pediatric care of sickle cell patients. Hematology, 2022(1), 442–449. https://doi.org/10.1182/hematology.2022000382
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Moore, A., Busch, M. P., Dziewulska, K., Francis, R. O., Hod, E. A., Zimring, J. C., D’Alessandro, A., & Page, G. P. (2022). Genome-wide metabolite quantitative trait loci analysis (mQTL) in red blood cells from volunteer blood donors. Journal of Biological Chemistry, 298(12), 102706. https://doi.org/10.1016/j.jbc.2022.102706
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Smaldone, A., Manwani, D., Aygun, B., Appiah-Kubi, A., Smith-Whitley, K., & Green, N. S. (2022). Assessing Multi-Level Barriers to Medication Adherence in Youth with Sickle Cell Disease Using Pharmacy-Based Prescription Refill Data. Blood, 140(Supplement 1), 2149–2150. https://doi.org/10.1182/blood-2022-157527
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Columbia Affiliation
Walters, M. C., Thompson, A. A., Kwiatkowski, J. L., Parikh, S., Mapara, M. Y., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Miller, A., Zhang, L., Chawla, A., Macari, E. R., Pierciey, F. J., Tisdale, J. F., & Kanter, J. (2022). Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study. Blood, 140(Supplement 1), 26–28. https://doi.org/10.1182/blood-2022-162288
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Zhang, C., Tremblay, D., Magnotti, J., Ronner, L., Podoltsev, N. A., Gotlib, J., Heaney, M. L., Kuykendall, A., O’Connell, C. L., Fleischman, A. G., Mesa, R., Yacoub, A., Mascarenhas, J., & Shammo, J. M. (2022). Exploratory Predictors for Hydroxyurea Treatment Duration in Polycythemia Vera: A Retrospective Multi-Institutional Database Analysis. Blood, 140(Supplement 1), 9693–9695. https://doi.org/10.1182/blood-2022-157873
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Platzbecker, U., Komrokji, R. S., Fenaux, P., Zeidan, A. M., Sekeres, M. A., Savona, M. R., Madanat, Y. F., Santini, V., Van Eygen, K., Raza, A., Germing, U., Berry, T., Dougherty, S., Shah, S., Sun, L., Huang, F., Feller, F., Wan, Y., Ikin, A., & Sherman, L. (2022). Imetelstat Achieved Prolonged, Continuous Transfusion Independence (TI) in Patients with Heavily Transfused Non-Del(5q) Lower-Risk Myelodysplastic Syndrome (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESAs) within the IMerge Phase 2 Study. Blood, 140(Supplement 1), 1106–1108. https://doi.org/10.1182/blood-2022-169050
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Locatelli, F., Lang, P., Li, A., Corbacioglu, S., de la Fuente, J., Wall, D. A., Liem, R., Meisel, R., Mapara, M. Y., Shah, A. J., Cappellini, M. D. D., Kattamis, A., Sheth, S., Bobruff, Y., Bower, L., Zhang, L., Sharma, A., Song, Y., Hobbs, W., & Frangoul, H. (2022). Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia. Blood, 140(Supplement 1), 4899–4901. https://doi.org/10.1182/blood-2022-166881
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George, S. A., Veludhandi, A., Xiang, Y., Stenger, E., Arnold, S. D., Mehta, A., Schirmer, D. A., Spencer, J. B., Guilcher, G. M., Bhatia, M., Abraham, A., Gomez-Lobo, V., Krishnamurti, L., & Meacham, L. R. (2022). Gonadal Hormone Production after Hematopoietic Cell Transplant (HCT) in Patients with Sickle Cell Disease (SCD): A Stellar Study. Blood, 140(Supplement 1), 1391–1392. https://doi.org/10.1182/blood-2022-168060
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Frangoul, H., Locatelli, F., Bhatia, M., Mapara, M. Y., Molinari, L., Sharma, A., Lobitz, S., de Montalembert, M., Rondelli, D., Steinberg, M., Walters, M. C., Imren, S., Zhang, L., Sharma, A., Song, Y., Simard, C., Hobbs, W., & Grupp, S. (2022). Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Severe Sickle Cell Disease. Blood, 140(Supplement 1), 29–31. https://doi.org/10.1182/blood-2022-162353
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Mishkin, A. D., Cheung, S. G., Hoffman, A., Leimbach, E. J., Dosovitz, S., & Mapara, M. (2022). High incidence of suicidal ideation in a series of patients with sickle cell disease after hematopoietic stem cell transplantation. Blood Advances, 6(19), 5542–5545. https://doi.org/10.1182/bloodadvances.2021006752
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P., B., R., I., R., O., D., M., E., M., P., K., N., G., & A., B. (2022). O-08: NEUROCOGNITIVE IMPAIRMENT IN UGANDAN CHILDREN WITH SICKLE CELL ANEMIA COMPARED TO NON-SICKLE SIBLINGS. HemaSphere, 6, 05–06. https://doi.org/10.1097/01.hs9.0000872840.49305.3f
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Kanter, J., Walters, M. C., Krishnamurti, L., Mapara, M. Y., Kwiatkowski, J. L., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Pierciey, F. J., Bonner, M., Miller, A., Zhang, X., Lynch, J., Kim, D., Ribeil, J.-A., Asmal, M., Goyal, S., Thompson, A. A., & Tisdale, J. F. (2022). Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease. New England Journal of Medicine, 386(7), 617–628. https://doi.org/10.1056/nejmoa2117175
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Roubinian, N. H., Reese, S. E., Qiao, H., Plimier, C., Fang, F., Page, G. P., Cable, R. G., Custer, B., Gladwin, M. T., Goel, R., Harris, B., Hendrickson, J. E., Kanias, T., Kleinman, S., Mast, A. E., Sloan, S. R., Spencer, B. R., Spitalnik, S. L., Busch, M. P., & Hod, E. A. (2022). Donor genetic and nongenetic factors affecting red blood cell transfusion effectiveness. JCI Insight, 7(1). https://doi.org/10.1172/jci.insight.152598
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Dei Zotti, F., Qiu, A., La Carpia, F., Moriconi, C., & Hudson, K. E. (2021). A New Murine Model of Primary Autoimmune Hemolytic Anemia (AIHA). Frontiers in Immunology, 12. https://doi.org/10.3389/fimmu.2021.752330
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Tremblay, D., Srisuwananukorn, A., Ronner, L., Podoltsev, N., Gotlib, J., Heaney, M. L., Kuykendall, A. T., O’Connell, C. L., Shammo, J. M., Fleischman, A., Mesa, R. A., Yacoub, A., Hoffman, R., Moshier, E., Zubizarreta, N., & Mascarenhas, J. (2021). European Leukemianet (ELN) Response Predicts Disease Progression but Not Thrombosis or Death in Polycythemia Vera (PV): An Analysis of a Multicenter Database. Blood, 138(Supplement 1), 240–240. https://doi.org/10.1182/blood-2021-148020
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Arnold, S. D., Bakshi, N., Chang, Z., Ross, D., Abraham, A., Bhatia, M., Guilcher, G. M. T., Haight, A., Hsieh, M., Kasow, K. A., Lane, P. A., Meacham, L., Olowoselu, F. O., & Krishnamurti, L. (2021). Long-Term Quality of Life after Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from the Sickle Cell Transplant Evaluation of Long Term and Late Effects Registry (STELLAR). Blood, 138(Supplement 1), 425–425. https://doi.org/10.1182/blood-2021-154280
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Askew, M., Smaldone, A., Gold, M. A., Smith-Whitley, K., Strouse, J. J., Jin, Z., & Green, N. S. (2021). National Survey of Pediatric Sickle Cell Providers on Their Contraceptive Practices for Female Patients. Blood, 138(Supplement 1), 1898–1898. https://doi.org/10.1182/blood-2021-154083
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Srisuwananukorn, A., Percha, B., Abu-Zeinah, G., Scandura, J., Krichevsky, S., Taylor, E., Silver, R. T., Thibaud, S., Ronner, L., Podoltsev, N., Gotlib, J., Heaney, M. L., Kuykendall, A. T., O’Connell, C., Shammo, J. M., Fleischman, A., Mesa, R. A., Yacoub, A., Hoffman, R., … Tremblay, D. (2021). Novel Machine Learning Algorithm Predicts Disease Progression in Polycythemia Vera (PV) with Readily-Available Baseline Characteristics. Blood, 138(Supplement 1), 2583–2583. https://doi.org/10.1182/blood-2021-150561
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Veludhandi, A., John, T. D., Bakshi, N., Arnold, S. D., Bhatia, M., Lane, P. A., Meacham, L., Khemani, K., Chaudhury, S., Olowoselu, F. O., Kasow, K. A., Hsieh, M., Abraham, A., Guilcher, G. M. T., & Krishnamurti, L. (2021). Mobility and Muscle Strength in Recipients of Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from Sickle Transplant Evaluation of Longterm and Late Effects Registry (STELLaR). Blood, 138(Supplement 1), 3030–3030. https://doi.org/10.1182/blood-2021-152383
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Santini, V., Fenaux, P., Van Eygen, K., Raza, A., Germing, U., Font, P., Diez-Campelo, M., Thepot, S., Vellenga, E., Patnaik, M. M., Jang, J. H., Sherman, L., Berry, T., Feller, F., Dougherty, S., Sun, L., Wan, Y., Rizo, A., Huang, F., & Platzbecker, U. (2021). On-Target Activity of Imetelstat Correlates with Clinical Benefits, Including Overall Survival (OS), in Heavily Transfused Non-Del(5q) Lower Risk MDS (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESAs). Blood, 138(Supplement 1), 2598–2598. https://doi.org/10.1182/blood-2021-148876
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Tisdale, J. F., Thompson, A. A., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Aygun, B., Kasow, K. A., Rifkin-Zenenberg, S., Schmidt, M., Pierciey, F. J., Whitney, D., Rogers, C., Nnamani, M., Foos, M., Miller, A., Zhang, X., Lynch, J., Walters, M. C., Kanter, J., & Bonner, M. (2021). Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT). Blood, 138(Supplement 1), 561–561. https://doi.org/10.1182/blood-2021-147760
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Walters, M. C., Tisdale, J. F., Mapara, M. Y., Krishnamurti, L., Kwiatkowski, J. L., Aygun, B., Kasow, K. A., Rifkin-Zenenberg, S., Jaroscak, J., Garbinsky, D., Chirila, C., Gallagher, M. E., Zhang, X., Ho, P.-R., Thompson, A. A., & Kanter, J. (2021). Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy. Blood, 138(Supplement 1), 7–7. https://doi.org/10.1182/blood-2021-146905
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