Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease: Lessons learned from the midpoint analysis

Deepa Manwani; Chunliang Xu; Sung Kyun Lee; George Amatuni; Hillel W. Cohen; Veronica Carullo; Kerry Morrone; Jennifer Davila; Patricia Ann Shi; Karen Ireland; Janine Keenan; Paul S. Frenette

doi:10.1016/j.ctim.2020.102481

Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease: Lessons learned from the midpoint analysis

Deepa Manwani, Chunliang Xu, Sung Kyun Lee, George Amatuni, Hillel W. Cohen, Veronica Carullo, Kerry Morrone, Jennifer Davila, Patricia Ann Shi, Karen Ireland, Janine Keenan, Paul S. Frenette

Research output: Contribution to journal › Article › peer-review

5 Scopus citations

Abstract

Sickle Cell Disease (SCD) is a chronic hemolytic disorder associated with frequent pain episodes, end organ damage and a shortened lifespan. Currently there exist no disease specific targeted therapies for the treatment of acute vaso-occlusive crisis (VOC) and management with analgesics and hydration is purely supportive. Improvement in understanding of disease pathophysiology has resulted in a great interest in disease modifying novel therapies and many are being evaluated in clinical trials. Here we report the results from the pre-specified mid-point analysis of the Phase 2 study of Intravenous Gamma Globulin (IVIG) for the treatment of acute VOC in patients with SCD and lessons learned.

Original language	English
Article number	102481
Journal	Complementary Therapies in Medicine
Volume	52
DOIs	https://doi.org/10.1016/j.ctim.2020.102481
State	Published - Aug 2020
Externally published	Yes

Keywords

IVIG
Immune globulin
Sickle

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10.1016/j.ctim.2020.102481

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Manwani, D., Xu, C., Lee, S. K., Amatuni, G., Cohen, H. W., Carullo, V., Morrone, K., Davila, J., Shi, P. A., Ireland, K., Keenan, J., & Frenette, P. S. (2020). Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease: Lessons learned from the midpoint analysis. Complementary Therapies in Medicine, 52, Article 102481. https://doi.org/10.1016/j.ctim.2020.102481

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title = "Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease: Lessons learned from the midpoint analysis",

abstract = "Sickle Cell Disease (SCD) is a chronic hemolytic disorder associated with frequent pain episodes, end organ damage and a shortened lifespan. Currently there exist no disease specific targeted therapies for the treatment of acute vaso-occlusive crisis (VOC) and management with analgesics and hydration is purely supportive. Improvement in understanding of disease pathophysiology has resulted in a great interest in disease modifying novel therapies and many are being evaluated in clinical trials. Here we report the results from the pre-specified mid-point analysis of the Phase 2 study of Intravenous Gamma Globulin (IVIG) for the treatment of acute VOC in patients with SCD and lessons learned.",

keywords = "IVIG, Immune globulin, Sickle",

author = "Deepa Manwani and Chunliang Xu and Lee, {Sung Kyun} and George Amatuni and Cohen, {Hillel W.} and Veronica Carullo and Kerry Morrone and Jennifer Davila and Shi, {Patricia Ann} and Karen Ireland and Janine Keenan and Frenette, {Paul S.}",

note = "Publisher Copyright: {\textcopyright} 2020 Elsevier Ltd",

year = "2020",

month = aug,

doi = "10.1016/j.ctim.2020.102481",

language = "English",

volume = "52",

journal = "Complementary Therapies in Medicine",

issn = "0965-2299",

publisher = "Churchill Livingstone",

}

Manwani, D, Xu, C, Lee, SK, Amatuni, G, Cohen, HW, Carullo, V, Morrone, K, Davila, J, Shi, PA, Ireland, K, Keenan, J & Frenette, PS 2020, 'Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease: Lessons learned from the midpoint analysis', Complementary Therapies in Medicine, vol. 52, 102481. https://doi.org/10.1016/j.ctim.2020.102481

Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease: Lessons learned from the midpoint analysis. / Manwani, Deepa; Xu, Chunliang; Lee, Sung Kyun et al.
In: Complementary Therapies in Medicine, Vol. 52, 102481, 08.2020.

Research output: Contribution to journal › Article › peer-review

TY - JOUR

T1 - Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease

T2 - Lessons learned from the midpoint analysis

AU - Manwani, Deepa

AU - Xu, Chunliang

AU - Lee, Sung Kyun

AU - Amatuni, George

AU - Cohen, Hillel W.

AU - Carullo, Veronica

AU - Morrone, Kerry

AU - Davila, Jennifer

AU - Shi, Patricia Ann

AU - Ireland, Karen

AU - Keenan, Janine

AU - Frenette, Paul S.

PY - 2020/8

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AB - Sickle Cell Disease (SCD) is a chronic hemolytic disorder associated with frequent pain episodes, end organ damage and a shortened lifespan. Currently there exist no disease specific targeted therapies for the treatment of acute vaso-occlusive crisis (VOC) and management with analgesics and hydration is purely supportive. Improvement in understanding of disease pathophysiology has resulted in a great interest in disease modifying novel therapies and many are being evaluated in clinical trials. Here we report the results from the pre-specified mid-point analysis of the Phase 2 study of Intravenous Gamma Globulin (IVIG) for the treatment of acute VOC in patients with SCD and lessons learned.

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KW - Immune globulin

KW - Sickle

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SN - 0965-2299

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ER -

Randomized phase 2 trial of Intravenous Gamma Globulin (IVIG) for the treatment of acute vaso-occlusive crisis in patients with sickle cell disease: Lessons learned from the midpoint analysis

Abstract

Keywords

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