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Start Over Author "Rothman, Jennifer A." Publisher ferrata storti foundation
4 results on '"Rothman, Jennifer A."'

1. Prevalence and management of iron overload in pyruvate kinase deficiency : report from the Pyruvate Kinase Deficiency Natural History Study

Author

van Beers, Eduard J., van Straaten, Stephanie, Morton, D. Holmes, Barcellini, Wilma, Eber, Stefan W., Glader, Bertil, Yaish, Hassan M., Chonat, Satheesh, Kwiatkowski, Janet L., Rothman, Jennifer A., Sharma, Mukta, Neufeld, Ellis J., Sheth, Sujit, Despotovic, Jenny M., Kollmar, Nina, Pospíšilová, Dagmar, Knoll, Christine M., Kuo, Kevin, Pastore, Yves D., Thompson, Alexis A., Newburger, Peter E., Ravindranath, Yaddanapudi, Wang, Winfred C., Wlodarski, Marcin W., Wang, Heng, Holzhauer, Susanne, Breakey, Vicky R., Verhovsek, Madeleine, Kunz, Joachim, McNaull, Melissa A., Rose, Melissa J., Bradeen, Heather A., Addonizio, Kathryn, Li, Anran, Al-Sayegh, Hasan, London, Wendy B., Grace, Rachael F., van Beers, Eduard J., van Straaten, Stephanie, Morton, D. Holmes, Barcellini, Wilma, Eber, Stefan W., Glader, Bertil, Yaish, Hassan M., Chonat, Satheesh, Kwiatkowski, Janet L., Rothman, Jennifer A., Sharma, Mukta, Neufeld, Ellis J., Sheth, Sujit, Despotovic, Jenny M., Kollmar, Nina, Pospíšilová, Dagmar, Knoll, Christine M., Kuo, Kevin, Pastore, Yves D., Thompson, Alexis A., Newburger, Peter E., Ravindranath, Yaddanapudi, Wang, Winfred C., Wlodarski, Marcin W., Wang, Heng, Holzhauer, Susanne, Breakey, Vicky R., Verhovsek, Madeleine, Kunz, Joachim, McNaull, Melissa A., Rose, Melissa J., Bradeen, Heather A., Addonizio, Kathryn, Li, Anran, Al-Sayegh, Hasan, London, Wendy B., and Grace, Rachael F.

Published
2019

2. Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study

Author

Poli VCK, UMC Utrecht, Poli Van Creveldkliniek Medisch, CDL Rode cel, Circulatory Health, van Beers, Eduard J., van Straaten, Stephanie, Morton, D. Holmes, Barcellini, Wilma, Eber, Stefan W., Glader, Bertil, Yaish, Hassan M., Chonat, Satheesh, Kwiatkowski, Janet L., Rothman, Jennifer A., Sharma, Mukta, Neufeld, Ellis J., Sheth, Sujit, Despotovic, Jenny M., Kollmar, Nina, Pospíšilová, Dagmar, Knoll, Christine M., Kuo, Kevin, Pastore, Yves D., Thompson, Alexis A., Newburger, Peter E., Ravindranath, Yaddanapudi, Wang, Winfred C., Wlodarski, Marcin W., Wang, Heng, Holzhauer, Susanne, Breakey, Vicky R., Verhovsek, Madeleine, Kunz, Joachim, McNaull, Melissa A., Rose, Melissa J., Bradeen, Heather A., Addonizio, Kathryn, Li, Anran, Al-Sayegh, Hasan, London, Wendy B., Grace, Rachael F., Poli VCK, UMC Utrecht, Poli Van Creveldkliniek Medisch, CDL Rode cel, Circulatory Health, van Beers, Eduard J., van Straaten, Stephanie, Morton, D. Holmes, Barcellini, Wilma, Eber, Stefan W., Glader, Bertil, Yaish, Hassan M., Chonat, Satheesh, Kwiatkowski, Janet L., Rothman, Jennifer A., Sharma, Mukta, Neufeld, Ellis J., Sheth, Sujit, Despotovic, Jenny M., Kollmar, Nina, Pospíšilová, Dagmar, Knoll, Christine M., Kuo, Kevin, Pastore, Yves D., Thompson, Alexis A., Newburger, Peter E., Ravindranath, Yaddanapudi, Wang, Winfred C., Wlodarski, Marcin W., Wang, Heng, Holzhauer, Susanne, Breakey, Vicky R., Verhovsek, Madeleine, Kunz, Joachim, McNaull, Melissa A., Rose, Melissa J., Bradeen, Heather A., Addonizio, Kathryn, Li, Anran, Al-Sayegh, Hasan, London, Wendy B., and Grace, Rachael F.

Published
2019

3. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.

Author

Rogers ZR, Nakano TA, Olson TS, Bertuch AA, Wang W, Gillio A, Coates TD, Chawla A, Castillo P, Kurre P, Gamper C, Bennett CM, Joshi S, Geddis AE, Boklan J, Nalepa G, Rothman JA, Huang JN, Kupfer GM, Cada M, Glader B, Walkovich KJ, Thompson AA, Hanna R, Vlachos A, Malsch M, Weller EA, Williams DA, and Shimamura A

Subjects
Anemia, Aplastic epidemiology, Anemia, Aplastic pathology, Antilymphocyte Serum adverse effects, Child, Preschool, Cyclosporine adverse effects, Female, Follow-Up Studies, Humans, Infant, Male, Retrospective Studies, United States epidemiology, Anemia, Aplastic drug therapy, Antilymphocyte Serum administration & dosage, Cyclosporine administration & dosage, Immunosuppression Therapy
Abstract

Quality of response to immunosuppressive therapy and long-term outcomes for pediatric severe aplastic anemia remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or refractory severe aplastic anemia for pediatric patients is also limited. The clinical features and outcomes for 314 children treated from 2002 to 2014 with immunosuppressive therapy for acquired severe aplastic anemia were analyzed retrospectively from 25 institutions in the North American Pediatric Aplastic Anemia Consortium. The majority of subjects (n=264) received horse anti-thymocyte globulin (hATG) plus cyclosporine (CyA) with a median 61 months follow up. Following hATG/CyA, 71.2% (95%CI: 65.3,76.6) achieved an objective response. In contrast to adult studies, the quality of response achieved in pediatric patients was high, with 59.8% (95%CI: 53.7,65.8) complete response and 68.2% (95%CI: 62.2,73.8) achieving at least a very good partial response with a platelet count ≥50×10 9 L. At five years post-hATG/CyA, overall survival was 93% (95%CI: 89,96), but event-free survival without subsequent treatment was only 64% (95%CI: 57,69) without a plateau. Twelve of 171 evaluable patients (7%) acquired clonal abnormalities after diagnosis after a median 25.2 months (range: 4.3-71 months) post treatment. Myelodysplastic syndrome or leukemia developed in 6 of 314 (1.9%). For relapsed/refractory disease, treatment with a hematopoietic stem cell transplant had a superior event-free survival compared to second immunosuppressive therapy treatment in a multivariate analysis (HR=0.19, 95%CI: 0.08,0.47; P =0.0003). This study highlights the need for improved therapies to achieve sustained high-quality remission for children with severe aplastic anemia., (Copyright© 2019 Ferrata Storti Foundation.)

Published
2019
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4. Prevalence and management of iron overload in pyruvate kinase deficiency: report from the Pyruvate Kinase Deficiency Natural History Study.

Author

van Beers EJ, van Straaten S, Morton DH, Barcellini W, Eber SW, Glader B, Yaish HM, Chonat S, Kwiatkowski JL, Rothman JA, Sharma M, Neufeld EJ, Sheth S, Despotovic JM, Kollmar N, Pospíšilová D, Knoll CM, Kuo K, Pastore YD, Thompson AA, Newburger PE, Ravindranath Y, Wang WC, Wlodarski MW, Wang H, Holzhauer S, Breakey VR, Verhovsek M, Kunz J, McNaull MA, Rose MJ, Bradeen HA, Addonizio K, Li A, Al-Sayegh H, London WB, and Grace RF

Subjects
Biomarkers, Blood Transfusion, DNA Mutational Analysis, Disease Management, Humans, Iron metabolism, Iron Overload diagnosis, Iron Overload epidemiology, Magnetic Resonance Imaging, Mutation, Prevalence, Iron Overload etiology, Iron Overload metabolism, Pyruvate Kinase deficiency
Published
2019
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