Resolution of Serious Vaso-occlusive Pain Crises: Results From The Ongoing Phase 1/2 HGB-206 Group C Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

被引:0
作者
Mapara, Markus [1 ]
Walters, Mark [2 ]
Thompson, Alexis [3 ,4 ]
Kwiatkowski, Janet [5 ,6 ]
Krishnamurti, Lakshmanan [7 ]
Aygun, Banu [8 ]
Kasow, Kimberly [9 ]
Rifkin-Zenenberg, Stacey [10 ]
Schmidt, Manfred [11 ]
Nnamani, Mauris [12 ]
VanNest, Sara [12 ]
Pierciey, Francis, Jr. [12 ]
Miller, Alex [12 ]
Chen, Ren [12 ]
Kim, Dennis [12 ]
Goyal, Sunita [12 ]
Kanter, Julie [13 ]
Tisdale, John [14 ]
机构
[1] Columbia Univ, Med Ctr, New York, NY USA
[2] UCSF Benioff Childrens Hosp Oakland, Oakland, CA USA
[3] Northwestern Univ, Feinberg Sch Med, Chicago, IL 60611 USA
[4] Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL USA
[5] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[6] Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA
[7] Emory Univ, Childrens Healthcare Atlanta, Atlanta, GA 30322 USA
[8] Cohen Childrens Med Ctr, Queens, NY USA
[9] Univ N Carolina, Chapel Hill, NC 27515 USA
[10] Hackensack Univ, Med Ctr, Hackensack, NJ USA
[11] GeneWerk GmbH, Heidelberg, Germany
[12] Bluebird Bio Inc, Cambridge, MA USA
[13] Univ Alabama Birmingham, Birmingham, AL USA
[14] NIH, Bldg 10, Bethesda, MD 20892 USA
关键词
D O I
暂无
中图分类号
Q6 [生物物理学];
学科分类号
071011 ;
摘要
O056
引用
收藏
页码:66 / 68
页数:3
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[21]   Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease [J].
Kanter, Julie ;
Walters, Mark C. ;
Hsieh, Matthew M. ;
Krishnamurti, Lakshmanan ;
Kwiatkowski, Janet ;
Kamble, Rammurti T. ;
von Kalle, Christof ;
Kuypers, Frans A. ;
Cavazzana, Marina ;
Leboulch, Philippe ;
Joseney-Antoine, Marcelyne ;
Asmal, Mohammed ;
Thompson, Alexis A. ;
Tisdale, John F. .
BLOOD, 2016, 128 (22)
[22]   Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease [J].
Kanter, Julie ;
Walters, Mark C. ;
Hsieh, Matthew ;
Krishnamurti, Lakshmanan ;
Kwiatkowski, Janet L. ;
Kamble, Rammurti ;
von Kalle, Christof ;
Joseney-Antoine, Marcelyne ;
Pierciey, Francis J., Jr. ;
Shi, Weiliang ;
Asmal, Mohammed ;
Thompson, Alexis A. ;
Tisdale, John F. .
BLOOD, 2017, 130
[23]   Etavopivat Reduces Incidence of Vaso-Occlusive Crises in Patients with Sickle Cell Disease: HIBISCUS Trial Phase 2 Results through 52 Weeks [J].
Delicou, Sophia ;
El Rassi, Fuad ;
Andemariam, Biree ;
Abboud, Miguel R. ;
Kanter, Julie ;
Telen, Marilyn J. ;
Githanga, Jessie ;
Inati, Adlette ;
Idris, Ibrahim ;
Navani, Sunil ;
Wu, Eric ;
Eisenberger, Andrew .
BLOOD, 2024, 144 :179-180
[24]   Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease [J].
Kanter, Julie ;
Walters, Mark C. ;
Hsieh, Matthew ;
Thompson, Alexis A. ;
Krishnamurti, Lakshmanan ;
Kwiatkowski, Janet ;
Kamble, Rammurti T. ;
von Kalle, Christof ;
Kuypers, Frans A. ;
Cavazzana, Marina ;
Leboulch, Philippe ;
Sandler, Laura ;
Soni, Sandeep ;
Tisdale, John F. .
BLOOD, 2015, 126 (23)
[25]   Mechanism(s)-of-Action for Arginine Therapy in Children with Sickle Cell Disease and Vaso-Occlusive Pain: Results of a Pharmacokinetics/Pharmacodynamics Study [J].
Korman, Rawan ;
Hatabah, Dunia ;
Brown, Lou Ann ;
Harris, Frank ;
Bakhsi, Nitya ;
Rees, Christopher ;
Griffiths, Mark ;
Leake, Deborah ;
Dampier, Carlton ;
Morris, Claudia R. .
BLOOD, 2023, 142
[26]   Randomized phase 2 trial of regadenoson for treatment of acute vaso-occlusive crises in sickle cell disease (vol 1, pg 1645, 2017) [J].
Field, J. J. ;
Majerus, E. ;
Gordeuk, V. R. .
BLOOD ADVANCES, 2017, 1 (23) :2058-2058
[27]   The Effect of Crizanlizumab on the Number of Days Requiring Opioid Use for Management of Pain Associated with Vaso-Occlusive Crises in Patients with Sickle Cell Disease: Results from the Sustain Trial [J].
Smith, Wally R. ;
Ataga, Kenneth I. ;
Saraf, Santosh L. ;
Adisa, Olufolake A. ;
Bailey, Miranda ;
Ramscar, Nicholas ;
Bonner, Ashley ;
Brown, Stephen ;
Pastor, Laura .
BLOOD, 2020, 136
[28]   Real-World Data on the Occurrence of Vaso-Occlusive Crises (VOCs) in Patients with Sickle Cell Disease (SCD) and a High Baseline Disease Burden Treated with Crizanlizumab: Results from a Managed Access Program (MAP) [J].
Cancado, Rodolfo D. ;
Colombatti, Raffaella ;
Quarta, Antonella ;
Arcioni, Francesco ;
De Bonnett, Laurie ;
Soliman, Wesam ;
Sarkar, Rajendra ;
Silva Pinto, Ana Cristina .
BLOOD, 2021, 138
[29]   Pharmacokinetics/Pharmacodynamics, Safety and Efficacy of Crizanlizumab in Patients with Sickle Cell Disease and a History of Vaso-Occlusive Crises: Results from the Phase II, Multicenter, Open-Label Solace-Adults Study [J].
Liles, Darla ;
Smith-Whitley, Kim ;
Brown, R. Clark ;
Shah, Nirmish ;
Nair, Santosh M. ;
Tanaka, Chiaki ;
Bodla, Shankaranand ;
Sanchez-Olle, Gessami ;
Albers, Urs ;
Kanter, Julie .
BLOOD, 2020, 136
[30]   An Analysis Of The Pediatric Sub-Group From The Phase 2 Study Of GMI 1070-A Novel Agent For The Vaso-Occlusive Crisis Of Sickle Cell Anemia [J].
McCavit, Timothy L. ;
Krishnamurti, Lakshmanan ;
Hsu, Lewis L. ;
Quinn, Charles T. ;
Odame, Isaac ;
Alvarez, Ofelia A. ;
Driscoll, Catherine ;
Smith-Whitley, Kim ;
Rhee, Seungshin ;
Wun, Ted ;
Telen, Marilyn J. ;
Thackray, Helen .
BLOOD, 2013, 122 (21)