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Volume 1, Issue 4

 

Cover Image

Cover Figure: Continuous improvement in relative survival rates of patients diagnosed with multiple myeloma in the United States between 1993 and 2012.
See the article by Costa et al.

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WASHINGTON, January 10,  2017 – Welcome to the “Advance Notice,”  newsletter which provides highlights from issues of Blood Advances, the open-access journal of the American Society of Hematology (ASH), that  are hand-picked by Blood Advances Editor-in-Chief Robert Negrin, MD.

Autosomal recessive p47phox-deficient chronic granulomatous disease (p47-CGD) is a serious immune disorder that results in life-threatening immunological deficiency. Correction of this disorder with gene modification is an attractive clinical approach. Merling et al explored strategies for correction of this monogenic disorder, using engineered zinc finger nucleases targeting the 2-nucleotide deletion at the start of exon 2 in induced pluripotent stem cells derived from patients with p47-CGD. Correction of this mutation resulted in production of p47phox and correction of oxidase activity. This paper documents an exciting approach that may someday become a clinical reality.

One of hematology's true success stories has been the multitude of treatments developed for patients with multiple myeloma. Costa et al discuss recent trends in its incidence and in the survival of patients with this life-threatening hematological disorder. Their report documents that there have been improvements in overall survival of patients <75 years of age, whereas older and minority patients unfortunately have not enjoyed similar improvements.

Featured Visual Abstract

Recent trends in multiple myeloma incidence and survival by age, race, and ethnicity in the United States

Luciano J. Costa, Ilene K. Brill, James Omel, Kelly Godby, Shaji K. Kumar and Elizabeth E. Brown

 

 

 

 

Blood Advances is the official open-access journal of the American Society of Hematology (ASH) (www.hematology.org), the world’s largest professional society concerned with the causes and treatment of blood disorders.

ASH’s mission is to further the understanding, diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems by promoting research, clinical care, education, training, and advocacy in hematology.