Advancing drug development in myelodysplastic syndromes

被引：0

作者：

Mina, Alain ^{[1
,2
]}

Mcgraw, Kathy L. ^{[1
,3
]}

Cunningham, Lea ^{[1
,2
]}

Kim, Nina ^{[4
]}

Jen, Emily Y. ^{[4
]}

Calvo, Katherine R. ^{[2
,5
]}

Ehrlich, Lori A. ^{[4
]}

Aplan, Peter D. ^{[2
,6
]}

Garcia-Manero, Guillermo ^{[7
]}

Foran, James M. ^{[8
]}

Garcia, Jacqueline S. ^{[9
]}

Zeidan, Amer M. ^{[10
,11
]}

Dezern, Amy E. ^{[12
]}

Komrokji, Rami ^{[13
]}

Sekeres, Mikkael A. ^{[14
]}

Scott, Bart ^{[15
]}

Buckstein, Rena ^{[16
]}

Tinsley-Vance, Sara ^{[13
]}

Verma, Amit ^{[17
]}

Wroblewski, Tanya ^{[4
]}

Paveletic, Steven ^{[1
,2
]}

Norsworthy, Kelly ^{[4
]}

机构：

[1] NCI, Immune Deficiency Cellular Therapy Program, Ctr Canc Res, NIH, Bethesda, MD USA

[2] NIH, Myeloid Malignancies Program, Bethesda, MD USA

[3] NCI, Lab Receptor Biol & Gene Express, NIH, Bethesda, MD USA

[4] US FDA, Ctr Drug Evaluat & Res, Silver Spring, MD USA

[5] Natl Inst Hlth, Dept Lab Med, Clin Ctr, Bethesda, MD USA

[6] NCI, Genet Branch, Ctr Canc Res, NIH, Bethesda, MD USA

[7] Univ Texas MD Anderson Canc Ctr, Dept Leukemia, Houston, TX USA

[8] Mayo Clin, Div Hematol, Jacksonville, FL USA

[9] Harvard Med Sch, Dana Farber Canc Inst, Dept Med Oncol, Boston, MA USA

[10] Yale Sch Med, Sect Hematol, Dept Internal Med, Yale Canc Ctr, New Haven, CT USA

[11] Yale Univ, Yale Comprehens Canc Ctr, New Haven, CT USA

[12] Johns Hopkins Univ Hosp, Sidney Kimmel Comprehens Canc Ctr, Div Hematol Malignancies, Baltimore, MD USA

[13] H Lee Moffitt Canc Ctr & Res Inst, Dept Malignant Hematol, Tampa, FL USA

[14] Univ Miami, Sylvester Comprehens Canc Ctr, Div Hematol, Miami, FL USA

[15] Fred Hutchinson Canc Res Ctr, Seattle, WA USA

[16] Sunnybrook Hlth Sci Ctr, Odette Canc Ctr, Toronto, ON, Canada

[17] Albert Einstein Coll Med, Dept Pathol, New York, NY USA

来源：

BLOOD ADVANCES | 2025年 / 9卷 / 05期

基金：

美国国家卫生研究院;

关键词：

QUALITY-OF-LIFE; HEALTH-ORGANIZATION CLASSIFICATION; INTERNATIONAL WORKING GROUP; PROGNOSTIC SCORING SYSTEM; RESPONSE CRITERIA; EPOETIN-ALPHA; OLDER-PEOPLE; OPEN-LABEL; MDS; DECITABINE;

D O I：

10.1182/bloodadvances.2024014865

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Myelodysplastic syndromes/neoplasms (MDSs) are heterogeneous stem cell malignancies characterized by poor prognosis and no curative therapies outside of allogeneic hematopoietic stem cell transplantation. Despite some recent approvals by the US Food and Drug Administration, (eg, luspatercept, ivosidenib, decitabine/cedazuridine, and imetelstat), there has been little progress in the development of truly transformative therapies for the treatment of patients with MDS. Challenges to advancing drug development in MDS are multifold but may be grouped into specific categories, including criteria for risk stratification and eligibility, response definitions, time-to-event end points, transfusion end points, functional assessments, and biomarker development. Strategies to address these challenges and optimize future clinical trial design for patients with MDS are presented here.

引用

页码：1095 / 1104

页数：10

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