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

来源：

BONE MARROW TRANSPLANTATION | 2021年 / 56卷 / SUPPL 1期

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

页码：66 / 68

页数：3

共 42 条

[21] Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease
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
[22] 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
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)
[23] Mechanism(s)-of-Action for Arginine Therapy in Children with Sickle Cell Disease and Vaso-Occlusive Pain: Results of a Pharmacokinetics/Pharmacodynamics Study
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
[24] Randomized phase 2 trial of regadenoson for treatment of acute vaso-occlusive crises in sickle cell disease (vol 1, pg 1645, 2017)
Field, J. J.
Majerus, E.
Gordeuk, V. R.
BLOOD ADVANCES, 2017, 1 (23) : 2058 - 2058
[25] 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
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
[26] 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)
Cancado, Rodolfo D.
Colombatti, Raffaella
Quarta, Antonella
Arcioni, Francesco
De Bonnett, Laurie
Soliman, Wesam
Sarkar, Rajendra
Silva Pinto, Ana Cristina
BLOOD, 2021, 138
[27] 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
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
[28] 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
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)
[29] Subdissociative intranasal ketamine plus standard pain therapy versus standard pain therapy in the treatment of paediatric sickle cell disease vaso-occlusive crises in resource-limited settings: study protocol for a randomised controlled trial
Young, James R.
Sawe, Hendry Robert
Mfinanga, Juma A.
Nshom, Ernest
Helm, Ethan
Moore, Charity G.
Runyon, Michael S.
Reynolds, Stacy L.
BMJ OPEN, 2017, 7 (07):
[30] Ticagrelor versus placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: Rationale and design of a randomized, double-blind, parallel-group, multicenter phase 3 study (HESTIA3)
Heeney, Matthew M.
Abboud, Miguel R.
Amilon, Carl
Andersson, Marielle
Githanga, Jessie
Inusa, Baba
Kanter, Julie
Leonsson-Zachrisson, Maria
Michelson, Alan D.
Berggren, Anders R.
CONTEMPORARY CLINICAL TRIALS, 2019, 85

← 1 2 3 4 5 →