Hematopoietic Stem-Cell Transplantation for Advanced Systemic Mastocytosis

被引：137

作者：

Ustun, Celalettin ^{[1
]}

Reiter, Andreas ^{[3
]}

Scott, Bart L. ^{[10
]}

Nakamura, Ryotaro ^{[11
]}

Damaj, Gandhi ^{[13
]}

Kreil, Sebastian ^{[3
]}

Shanley, Ryan ^{[1
]}

Hogan, William J. ^{[2
]}

Perales, Miguel-Angel ^{[20
]}

Shore, Tsiporah ^{[21
]}

Baurmann, Herrad ^{[4
]}

Stuart, Robert ^{[22
]}

Gruhn, Bernd ^{[5
]}

Doubek, Michael ^{[23
]}

Hsu, Jack W. ^{[24
]}

Tholouli, Eleni ^{[25
]}

Gromke, Tanja ^{[6
]}

Godley, Lucy A. ^{[26
]}

Pagano, Livio ^{[27
]}

Gilman, Andrew ^{[29
]}

Wagner, Eva Maria ^{[7
]}

Shwayder, Tor ^{[30
]}

Bornhaeuser, Martin ^{[8
]}

Papadopoulos, Esperanza B. ^{[20
]}

Boehm, Alexandra ^{[31
]}

Vercellotti, Gregory ^{[1
]}

Van Lint, Maria Teresa ^{[28
]}

Schmid, Christoph ^{[9
]}

Rabitsch, Werner ^{[32
]}

Pullarkat, Vinod ^{[12
]}

Legrand, Faezeh ^{[14
,15
]}

Yakoub-agha, Ibrahim ^{[16
]}

Saber, Wael ^{[33
]}

Barrett, John ^{[34
]}

Hermine, Olivier ^{[17
,18
,19
]}

Hagglund, Hans ^{[35
]}

Sperr, Wolfgang R. ^{[32
]}

Popat, Uday ^{[36
]}

Alyea, Edwin P. ^{[37
]}

Devine, Steven ^{[38
]}

Deeg, H. Joachim ^{[10
]}

Weisdorf, Daniel ^{[1
]}

Akin, Cem ^{[37
]}

Valent, Peter ^{[32
]}

机构：

[1] Univ Minnesota, Minneapolis, MN 55455 USA

[2] Mayo Clin, Rochester, MN USA

[3] Univ Med Mannheim, Mannheim, Germany

[4] Stiftung Deutsch Klin Diagnost, Wiesbaden, Germany

[5] Jena Univ Hosp, Jena, Germany

[6] Univ Essen Gesamthsch, Essen, Germany

[7] Univ Med Mainz, Mainz, Germany

[8] Univ Klinikum Dresden, Dresden, Germany

[9] Klinikum Augsburg, Augsburg, Germany

[10] Univ Washington, Seattle, WA 98195 USA

[11] City Hope Natl Med Ctr, Duarte, CA 91010 USA

[12] Univ So Calif, Los Angeles, CA USA

[13] Univ Caen, Fac Med, F-14032 Caen, France

[14] Nice Univ Hosp, Nice, France

[15] SFGM TC, Lille, France

[16] CHU Lille, F-59037 Lille, France

[17] Univ Paris 05, Hop Necker Enfants Malad, AP HP, Fac Med, Paris, France

[18] AP HP Necker Enfants Malades, Paris, France

[19] Ctr Reference Mastocytoses, Paris, France

[20] Mem Sloan Kettering Canc Ctr, New York, NY 10021 USA

[21] New York Presbyterian Hosp, Weill Cornell Med Ctr, New York, NY 10021 USA

[22] Med Univ S Carolina, Charleston, SC 29425 USA

[23] Masaryk Univ, Brno, Czech Republic

[24] Univ Florida, Gainesville, FL USA

[25] Manchester Royal Infirm, Manchester M13 9WL, Lancs, England

[26] Univ Chicago, Chicago, IL 60637 USA

[27] Univ Cattolica Sacro Cuore, I-20123 Milan, Italy

[28] Ist Ricovero & Cura Carattere Sci San Martino IST, Genoa, Italy

[29] Levine Childrens Hosp, Charlotte, NC USA

[30] Henry Ford Hosp, Detroit, MI 48202 USA

[31] Elisabethinen Hosp, Linz, Austria

[32] Med Univ Vienna, Vienna, Austria

[33] Med Coll Wisconsin, Milwaukee, WI 53226 USA

[34] NHLBI, Bethesda, MD 20892 USA

[35] Karolinska Univ Hosp, Stockholm, Sweden

[36] Univ Texas MD Anderson Canc Ctr, Houston, TX 77030 USA

[37] Harvard Univ, Sch Med, Brigham & Womens Hosp, Boston, MA 02115 USA

[38] Ohio State Univ, Columbus, OH 43210 USA

来源：

JOURNAL OF CLINICAL ONCOLOGY | 2014年 / 32卷 / 29期

基金：

美国国家卫生研究院;

关键词：

ACUTE MYELOID-LEUKEMIA; NEOPLASTIC MAST-CELLS; TYROSINE KINASE INHIBITORS; CHROMOSOME-NEGATIVE ACUTE; MINIMAL RESIDUAL DISEASE; C-KIT MUTATIONS; LINEAGE DISEASE; PROGNOSTIC IMPACT; CYTOGENETIC ABNORMALITIES; MARROW TRANSPLANTATION;

D O I：

10.1200/JCO.2014.55.2018

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Purpose Advanced systemic mastocytosis (SM), a fatal hematopoietic malignancy characterized by drug resistance, has no standard therapy. The effectiveness of allogeneic hematopoietic stem-cell transplantation (alloHCT) in SM remains unknown. Patients and Methods In a global effort to define the value of HCT in SM, 57 patients with the following subtypes of SM were evaluated: SM associated with clonal hematologic non-mast cell disorders (SM-AHNMD; n = 38), mast cell leukemia (MCL; n = 12), and aggressive SM (ASM; n = 7). Median age of patients was 46 years (range, 11 to 67 years). Donors were HLA-identical (n = 34), unrelated (n = 17), umbilical cord blood (n = 2), HLA-haploidentical (n = 1), or unknown (n = 3). Thirty-six patients received myeloablative conditioning (MAC), and 21 patients received reduced-intensity conditioning (RIC). Results Responses in SM were observed in 40 patients (70%), with complete remission in 16 patients (28%). Twelve patients (21%) had stable disease, and five patients (9%) had primary refractory disease. Overall survival (OS) at 3 years was 57% for all patients, 74% for patients with SM-AHNMD, 43% for those with ASM, and 17% for those with MCL. The strongest risk factor for poor OS was MCL. Survival was also lower in patients receiving RIC compared with MAC and in patients having progression compared with patients having stable disease or response. Conclusion AlloHCT was associated with long-term survival in patients with advanced SM. Although alloHCT may be considered as a viable and potentially curative therapeutic option for advanced SM in the meantime, given that this is a retrospective analysis with no control group, the definitive role of alloHCT will need to be determined by a prospective trial. (C) 2014 by American Society of Clinical Oncology

引用

页码：3264 / +

页数：18

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