Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Comment
. 2018 Jan 11;131(2):263-265.
doi: 10.1182/blood-2017-06-790121. Epub 2017 Nov 2.

Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study

Ali T Taher  1 , Zeynep Karakas  2 , Elena Cassinerio  3 , Noppadol Siritanaratkul  4 , Antonis Kattamis  5 , Aurelio Maggio  6 , Stefano Rivella  7 , Norbert Hollaender  8 , Bruyère Mahuzier  9 , Brian Gadbaw  10 , Yesim Aydinok  11
Affiliations
Comment

Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study

Ali T Taher et al. Blood. .
No abstract available

PubMed Disclaimer

Conflict of interest statement

Conflict-of-interest disclosure: A.T.T. received honoraria and research funding from Novartis and research funding from Celgene and Roche. Z.K. received research funding from Novartis. N.S. received research funding from Novartis, Janssen-Cilag, Roche, and Pfizer. A.K. received honoraria and research funding from Novartis, holds Novartis equity ownership, and received honoraria from ApoPharma. S.R. held membership on an entity's Board of Directors or advisory committees of Ionis Pharmaceutical, received research funding from Ionis Pharmaceutical, and provides consultancy to Ionis Pharmaceutical. N.H., B.M., and B.G. are employed by Novartis. Y.A. held membership on an entity's Board of Directors or advisory committees of Novartis and received research funding and Speakers Bureau, and received research funding from Shire, Celgene, and Cerus. The remaining authors declare no competing financial interests.

Figures

Figure 1.
Figure 1.
Scatter-plot of hematocrit-adjusted volume per 4 weeks at on treatment vs pretreatment periods. Each circle corresponds to 1 patient. The diagonal line indicates equal hematocrit-adjusted volume on treatment (weeks 6-30) and at baseline; dotted lines show 10% of decrease or increase.
Figure 2.
Figure 2.
Spleen response in transfusion-dependent thalassemic patients treated with ruxolitinib. (A) Change in spleen length from baseline below the left costal margin over time. (B) Percent change in spleen volume from baseline at week 30 measured by magnetic resonance imaging. Max, maximum; min, minimum.
See this image and copyright information in PMC

Comment on

References

    1. Melchiori L, Gardenghi S, Rivella S. β-thalassemia: HiJAKing ineffective erythropoiesis and iron overload. Adv Hematol. 2010;2010(938640):938640. - PMC - PubMed
    1. Makis A, Hatzimichael E, Papassotiriou I, Voskaridou E. 2017 clinical trials update in new treatments of β-thalassemia. Am J Hematol. 2016;91(11):1135-1145. - PubMed
    1. Rivella S. The role of ineffective erythropoiesis in non-transfusion-dependent thalassemia. Blood Rev. 2012;26(suppl 1):S12-S15. - PMC - PubMed
    1. Kuhrt D, Wojchowski DM. Emerging EPO and EPO receptor regulators and signal transducers. Blood. 2015;125(23):3536-3541. - PMC - PubMed
    1. Libani IV, Guy EC, Melchiori L, et al. Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia. Blood. 2008;112(3):875-885. - PMC - PubMed