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Mannuronic Acid in Low‐Risk and Intermediate‐1‐Risk Myelodysplastic Syndromes
Author(s) -
Ghaderi Afshin,
Nodehi Sayyed Reza Safaee,
Bakhtiari Tahereh,
Aslani Mona,
Aghazadeh Zahra,
Matsuo Hidenori,
Rehm Bernd H. A.,
Cuzzocrea Salvatore,
Mirshafiey Abbas
Publication year - 2020
Publication title -
the journal of clinical pharmacology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.92
H-Index - 116
eISSN - 1552-4604
pISSN - 0091-2700
DOI - 10.1002/jcph.1587
Subject(s) - tolerability , medicine , myelodysplastic syndromes , adverse effect , rheumatoid arthritis , international prognostic scoring system , clinical trial , clinical endpoint , bone marrow suppression , gastroenterology , bone marrow , toxicity
Abstract The discovery of hematologic improvement and bone marrow modification by the drug β‐D mannuronic acid (M2000) during treatment of rheumatoid arthritis in phase 1/2/3 clinical trials prompted us to design a new trial to target hematologic deficits in myelodysplastic syndromes (MDS). In this open‐label, randomized phase 2 clinical trial, the potential effect and tolerability of drug M2000 was assessed in patients with low‐ and intermediate‐1‐risk MDS. The primary efficacy end point was hematologic improvement after 12 weeks of β‐D‐mannuronic acid therapy. Among 34 enrolled patients, half received their conventional therapy plus β‐D‐mannuronic acid, and the other half received only conventional drugs. In the conventional + β‐D mannuronic acid treatment group, hematologic improvement and development of transfusion independence and/or reduction in transfusion requirements were seen in 12 patients (92.3%) and 1 patient (7.7%), respectively. Moreover, 5 patients (38.5%), 2 patients (15.4%), and 1 patient (7.7%) in the β‐D‐mannuronic acid‐treated group showed hematologic improvement of the major parameters of erythroid, neutrophil, and platelet responses, respectively, based on the International Working Group criteria), whereas in the conventional treatment group as control, no hematologic improvements including erythroid, neutrophil, and platelet response was seen. In this trial, the addition of β‐D mannuronic acid to conventional treatment showed promising results in MDS patients with low and intermediate‐1 risk with effects on hematologic improvements without significant adverse effect.