Myelodysplastic Syndromes
December 2017 Issue

Nearly every major medical breakthrough over the past 50 years can be attributed to new discoveries that were made possible through meticulous biomedical research. Many diseases and conditions that previously took a heavy toll on patients in terms of both morbidity and mortality are now either treatable or preventable thanks in large part to painstaking research that expanded the frontiers of scientific knowledge and moved the most promising basic research developments into tangible health benefits through human clinical trails. Ongoing research is also the key that will open the door to additional major medical advances and breakthroughs in the future.

Our staff at Medifocus is committed to keeping our subscribers to the Medifocus Digest Alert on Myelodysplastic Syndromes abreast of the latest new research developments that have recently been published in the medical literature for this condition. In this issue of the Digest Alert, you will find a focused list of hand-picked journal article references that represent the latest advances in basic and clinical research for Myelodysplastic Syndromes. These articles represent the current state-of-the-art of the research that will hopefully lead to additional major advances and breakthroughs in the clinical management of Myelodysplastic Syndromes. You can access the summaries of each article referenced below by simply clicking on the article Title.

We will continue to update you with the latest scientific and clinical developments through the Medifocus Digest Alert on Myelodysplastic Syndromes every 3 months. Thank you for your ongoing subscription to this publication.

Elliot Jacob, Ph.D.
Vice-President - Content, Inc.

1:Myelodysplastic Syndrome, Unclassifiable (MDS-U) With 1% Blasts Is a Distinct Subgroup of MDS-U With a Poor Prognosis.
Authors:Margolskee E, Hasserjian RP, Hassane D, Tam W, Mathew S, Ok CY, Wang SA, Oak J, Arber DA, Orazi A
Institution:Department of Pathology and Laboratory Medicine, Weill Cornell Medical College/New York Presbyterian Hospital, New York, NY.; Department of Pathology, Massachusetts General Hospital, Boston.; Department of Hematopathology, University of Texas M.D. Anderson Cancer Center, Houston.; Department of Pathology, Stanford University Medical Center, Stanford, CA.
Journal:Am J Clin Pathol. 2017 Jul 1;148(1):49-57. doi: 10.1093/ajcp/aqx043.

2:Luspatercept for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes (PACE-MDS): a multicentre, open-label phase 2 dose-finding study with long-term extension study.
Authors:Platzbecker U, Germing U, Gotze KS, Kiewe P, Mayer K, Chromik J, Radsak M, Wolff T, Zhang X, Laadem A, Sherman ML, Attie KM, Giagounidis A
Institution:Universitatsklinikum "Carl Gustav Carus" der Technischen Universitat Dresden, Dresden, Germany. Electronic address: Dusseldorf, Dusseldorf, Germany. Germany.
Journal:Lancet Oncol. 2017 Oct;18(10):1338-1347. doi: 10.1016/S1470-2045(17)30615-0. Epub 2017 Sep 1.

3:Observational Monitoring of Patients with Aplastic Anemia and Low/Intermediate-1 Risk of Myelodysplastic Syndromes Complicated with Iron Overload.
Authors:Du Y, Long Z, Chen M, Han B, Hou B, Feng F
Institution:Department of Hematology, Chinese Academy of Medical Sciences, Peking Union Medical College, Peking Union Medical College Hospital, Beijing, China.
Journal:Acta Haematol. 2017;138(2):119-128. doi: 10.1159/000479422. Epub 2017 Sep 2.

4:A comparison of therapeutic dosages of decitabine in treating myelodysplastic syndrome: a meta-analysis.
Authors:Yang B, Yu R, Cai L, Chi X, Liu C, Yang L, Wang X, He P, Lu X
Institution:Department of Geriatric Hematology, Nanlou Clinic, Chinese PLA General Hospital, Fuxing Road 28, Handian District, Beijing, 100853, China. University, Beijing, 100038, China. Hospital, Beijing, 100853, China. 100800, China. Beijing, 100853, China. China. University, Beijing, 100038, China. China. Fuxing Road 28, Handian District, Beijing, 100853, China.
Journal:Ann Hematol. 2017 Nov;96(11):1811-1823. doi: 10.1007/s00277-017-3102-y. Epub 2017 Aug 25.

5:Encouraging results with the compassionate use of hydralazine/valproate (TRANSKRIP) as epigenetic treatment for myelodysplastic syndrome (MDS).
Authors:Candelaria M, Burgos S, Ponce M, Espinoza R, Duenas-Gonzalez A
Institution:Division de Investigacion Clinica, Instituto Nacional de Cancerologia, Av. San Fernando 22, Tlalpan, 14080, Mexico, D.F., Mexico. Fernando 22, Tlalpan, 14080, Mexico, D.F., Mexico. Fernando 22, Tlalpan, 14080, Mexico, D.F., Mexico. Biomedicas (IIB), Instituto Nacional de Cancerologia (INCan), Universidad Nacional Autonoma de Mexico (UNAM), Mexico, Mexico.
Journal:Ann Hematol. 2017 Nov;96(11):1825-1832. doi: 10.1007/s00277-017-3103-x. Epub 2017 Aug 23.

6:Benefits of hypomethylating therapy in IPSS lower-risk myelodysplastic syndrome patients: A retrospective multicenter case series study.
Authors:Lee JH, Kim YJ, Sohn SK, Yoon SS, Kim H, Cheong JW, Lee WS, Lee GW, Lim SN, Kim MK, Lee HS, Kim HJ
Institution:Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea. Electronic address:; Department of Hematology, Cancer Research Institute, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea.; Department of Hematology-Oncology, Kyungpook National University Hospital, Daegu, Republic of Korea.; Department of Internal Medicine, Seoul National University Hospital, Seoul National University College of Medicine, Seoul, Republic of Korea.; Department of Hematology and Oncology, Ulsan University Hospital, University of Ulsan College of Medicine, Ulsan, Republic of Korea.
Journal:Leuk Res. 2017 Sep;60:135-144. doi: 10.1016/j.leukres.2017.08.004. Epub 2017 Aug 15.

7:Early treatment initiation in lower-risk myelodysplastic syndromes produces an earlier and higher rate of transfusion independence.
Authors:Cogle CR, Reddy SR, Chang E, Papoyan E, Broder MS, McGuire M, Binder G
Institution:Division of Hematology and Oncology, Department of Medicine, College of Medicine, University of Florida, 1600 SW Archer Rd, Box 100278, Gainesville, FL 32610, USA. Electronic address: Electronic address: Electronic address: Electronic address: Electronic address: Electronic address:
Journal:Leuk Res. 2017 Sep;60:123-128. doi: 10.1016/j.leukres.2017.07.008. Epub 2017 Aug 1.

8:Randomized phase 2 study of low-dose decitabine vs low-dose azacitidine in lower-risk MDS and MDS/MPN.
Authors:Jabbour E, Short NJ, Montalban-Bravo G, Huang X, Bueso-Ramos C, Qiao W, Yang H, Zhao C, Kadia T, Borthakur G, Pemmaraju N, Sasaki K, Estrov Z, Cortes J, Ravandi F, Alvarado Y, Komrokji R, Sekeres MA, Steensma DP, DeZern A, Roboz G, Kantarjian H, Garcia-Manero G
Institution:The University of Texas MD Anderson Cancer Center, Houston, TX.
Journal:Blood. 2017 Sep 28;130(13):1514-1522. doi: 10.1182/blood-2017-06-788497. Epub 2017 Aug 3.

9:Smoking and alcohol and subsequent risk of myelodysplastic syndromes in Japan: the Japan Public Health Centre-based Prospective Study.
Authors:Ugai T, Matsuo K, Sawada N, Iwasaki M, Yamaji T, Shimazu T, Sasazuki S, Inoue M, Kanda Y, Tsugane S
Institution:Division of Molecular and Clinical Epidemiology, Aichi Cancer Centre Research Institute, Nagoya, Japan. Saitama, Japan.
Journal:Br J Haematol. 2017 Sep;178(5):747-755. doi: 10.1111/bjh.14749. Epub 2017 Jul 17.

10:Hypomethylating agents (HMA) treatment for myelodysplastic syndromes: alternatives in the frontline and relapse settings.
Authors:Uy N, Singh A, Gore SD, Prebet T
Institution:a Section of Hematology, Department of Internal Medicine , Yale School of Medicine , New Haven , CT , USA. Medicine , New Haven , CT , USA. Medicine , New Haven , CT , USA.
Journal:Expert Opin Pharmacother. 2017 Aug;18(12):1213-1224. doi: 10.1080/14656566.2017.1349100. Epub 2017 Aug 1.

11:A phase 3 randomized placebo-controlled trial of darbepoetin alfa in patients with anemia and lower-risk myelodysplastic syndromes.
Authors:Platzbecker U, Symeonidis A, Oliva EN, Goede JS, Delforge M, Mayer J, Slama B, Badre S, Gasal E, Mehta B, Franklin J
Institution:University Hospital Carl Gustav Carus Dresden, Medizinische Klinik und Poliklinik I, Dresden, Germany. Medical School, Patras, Greece. Calabria, Italy. Switzerland. Leuven, Leuven, Belgium. and Faculty of Medicine, Masaryk University, Brno, Czech Republic. Avignon, France.
Journal:Leukemia. 2017 Sep;31(9):1944-1950. doi: 10.1038/leu.2017.192. Epub 2017 Jun 19.

12:Role of serum ferritin level on overall survival in patients with myelodysplastic syndromes: Results of a meta-analysis of observational studies.
Authors:Pileggi C, Di Sanzo M, Mascaro V, Marafioti MG, Costanzo FS, Pavia M
Institution:Department of Health Sciences, University of Catanzaro 'Magna Graecia', Catanzaro, Italy.; Department of Experimental and Clinical Medicine, University of Catanzaro 'Magna Graecia', Catanzaro, Italy.
Journal:PLoS One. 2017 Jun 16;12(6):e0179016. doi: 10.1371/journal.pone.0179016. eCollection 2017.

13:Romiplostim monotherapy in thrombocytopenic patients with myelodysplastic syndromes: long-term safety and efficacy.
Authors:Fenaux P, Muus P, Kantarjian H, Lyons RM, Larson RA, Sekeres MA, Becker PS, Orejudos A, Franklin J
Institution:Service d'Hematologie Clinique, Hopital St. Louis and Paris 7 University, Paris, France. Netherlands. Houston, TX, USA. USA.
Journal:Br J Haematol. 2017 Sep;178(6):906-913. doi: 10.1111/bjh.14792. Epub 2017 Jun 14.

14:Individual outcome prediction for myelodysplastic syndrome (MDS) and secondary acute myeloid leukemia from MDS after allogeneic hematopoietic cell transplantation.
Authors:Heuser M, Gabdoulline R, Loffeld P, Dobbernack V, Kreimeyer H, Pankratz M, Flintrop M, Liebich A, Klesse S, Panagiota V, Stadler M, Wichmann M, Shahswar R, Platzbecker U, Thiede C, Schroeder T, Kobbe G, Geffers R, Schlegelberger B, Gohring G, Kreipe HH, Germing U, Ganser A, Kroger N, Koenecke C, Thol F
Institution:Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Carl-Neuberg Str. 1, 30625, Hannover, Germany.; Medizinische Klinik und Poliklinik I, Universtitatsklinikum Carl Gustav Carus, Dresden, Germany.; Klinik fur Hamatologie, Onkologie und Klinische Immunologie, Heinrich Heine University, Dusseldorf, Germany.; Helmholtz Centre for Infection Research, Braunschweig, Germany.; Institute of Human Genetics, Hannover Medical School, Hannover, Germany.
Journal:Ann Hematol. 2017 Aug;96(8):1361-1372. doi: 10.1007/s00277-017-3027-5. Epub 2017 Jun 13.

15:Success in bone marrow failure? Novel therapeutic directions based on the immune environment of myelodysplastic syndromes.
Authors:Cull AH, Rauh MJ
Institution:Department of Pathology and Molecular Medicine, Queen's University, Kingston, Ontario, Canada. Ontario, Canada
Journal:J Leukoc Biol. 2017 Aug;102(2):209-219. doi: 10.1189/jlb.5RI0317-083R. Epub 2017 Jun 8.

16:Randomized Phase II Study of Azacitidine Alone or in Combination With Lenalidomide or With Vorinostat in Higher-Risk Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia: North American Intergroup Study SWOG S1117.
Authors:Sekeres MA, Othus M, List AF, Odenike O, Stone RM, Gore SD, Litzow MR, Buckstein R, Fang M, Roulston D, Bloomfield CD, Moseley A, Nazha A, Zhang Y, Velasco MR, Gaur R, Atallah E, Attar EC, Cook EK, Cull AH, Rauh MJ, Appelbaum FR, Erba HP
Institution:Mikkael A. Sekeres and Aziz Nazha, Cleveland Clinic, Cleveland; Clara D. Bloomfield, The Ohio State University Comprehensive Cancer Center, Columbus, OH; Megan Othus and Anna Moseley, SWOG Statistical Center; Min Fang and Frederick R. Appelbaum, Fred Hutchinson Cancer Research Center, Seattle, WA; Alan F. List, H. Lee Moffitt Cancer Center, Tampa, FL; Olatoyosi Odenike, University of Chicago, Chicago; Mario R. Velasco, Heartland NCORP/Cancer Care Specialists of Central Illinois, Decatur, IL; Richard M. Stone, Dana Farber Cancer Institute; Eyal C. Attar, Massachusetts General Hospital, Boston, MA; Steven D. Gore, Yale University, New Haven, CT; Mark R. Litzow, Mayo Clinic, Rochester, MN; Rena Buckstein, Sunnybrook Health Sciences Centre, Toronto; Elina K. Cook, Alyssa H. Cull, and Michael J. Rauh, Queen's University, Kingston, Ontario, Canada; Diane Roulston, University of Michigan, Ann Arbor, MI; Yanming Zhang, Memorial Sloan-Kettering Cancer Center, New York, NY; Rakesh Gaur, Kansas City National Cancer Institute Community Oncology Research Program, Prairie Village, KS; Ehab Atallah, Medical College of Wisconsin, Milwaukee, WI; and Harry P. Erba, University of Alabama, Birmingham, AL.
Journal:J Clin Oncol. 2017 Aug 20;35(24):2745-2753. doi: 10.1200/JCO.2015.66.2510. Epub 2017 May 9.

17:Iron-chelating therapy with deferasirox in transfusion-dependent, higher risk myelodysplastic syndromes: a retrospective, multicentre study.
Authors:Musto P, Maurillo L, Simeon V, Poloni A, Finelli C, Balleari E, Ricco A, Rivellini F, Cortelezzi A, Tarantini G, Villani O, Mansueto G, Milella MR, Scapicchio D, Marziano G, Breccia M, Niscola P, Sanna A, Clissa C, Voso MT, Fenu S, Venditti A, Santini V, Angelucci E, Levis A
Institution:Scientific Direction, IRCCS-CROB, 'Referral Cancer Centre of Basilicata', Rionero In Vulture (Pz), Italy.; Haematology, Department of Biomedicine and Prevention, 'Tor Vergata' University, Rome, Italy.; Laboratory of Pre-clinical and Translational Research, IRCCS-CROB, 'Referral Cancer Centre of Basilicata', Rionero In Vulture (Pz), Italy.; Haematology Clinic, Department of Clinic and Molecular Sciences, 'Universita Politecnica delle Marche', Ancona, Italy.; 'Seragnoli Institute of Haematology', University School of Medicine, Bologna, Italy.
Journal:Br J Haematol. 2017 Jun;177(5):741-750. doi: 10.1111/bjh.14621. Epub 2017 Apr 17.

18:A phase 1 study of the CXCR4 antagonist plerixafor in combination with high-dose cytarabine and etoposide in children with relapsed or refractory acute leukemias or myelodysplastic syndrome: A Pediatric Oncology Experimental Therapeutics Investigators' Consortium study (POE 10-03).
Authors:Cooper TM, Sison EAR, Baker SD, Li L, Ahmed A, Trippett T, Gore L, Macy ME, Narendran A, August K, Absalon MJ, Boklan J, Pollard J, Magoon D, Brown PA
Institution:Seattle Children's Cancer and Blood Disorders Center, University of Washington, Seattle, Washington. Houston, Texas. University School of Medicine, Baltimore, Maryland. University School of Medicine, Baltimore, Maryland.
Journal:Pediatr Blood Cancer. 2017 Aug;64(8). doi: 10.1002/pbc.26414. Epub 2017 Apr 14.

19:Comparison of clinical outcomes and prognostic utility of risk stratification tools in patients with therapy-related vs de novo myelodysplastic syndromes: a report on behalf of the MDS Clinical Research Consortium.
Authors:Zeidan AM, Al Ali N, Barnard J, Padron E, Lancet JE, Sekeres MA, Steensma DP, DeZern A, Roboz G, Jabbour E, Garcia-Manero G, List A, Komrokji R
Institution:Section of Hematology, Department of Internal Medicine, Yale University, and Yale Comprehensive Cancer Center, New Haven, CT, USA. Institute, Tampa, FL, USA.
Journal:Leukemia. 2017 Jun;31(6):1391-1397. doi: 10.1038/leu.2017.33. Epub 2017 Jan 23.

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