Allogeneic Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for Treatment of Marrow Failure Disorders

被引：45

作者：

Burroughs, Lauri M. ^{[1
,2
,3
,4
]}

Shimamura, Akiko ^{[1
,2
,3
,4
]}

Talano, Julie-An ^{[5
,6
]}

Domm, Jennifer A. ^{[7
]}

Baker, Kelsey K. ^{[1
]}

Delaney, Colleen ^{[1
,2
,3
,4
]}

Frangoul, Haydar ^{[7
]}

Margolis, David A. ^{[5
,6
]}

Baker, K. Scott ^{[1
,2
,3
,4
]}

Nemecek, Eneida R. ^{[8
]}

Geddis, Amy E. ^{[2
,3
,4
]}

Sandmaier, Brenda M. ^{[1
,2
,3
]}

Deeg, H. Joachim ^{[1
,2
,3
]}

Storb, Rainer ^{[1
,2
,3
]}

Woolfrey, Ann E. ^{[1
,2
,3
,4
]}

机构：

[1] Fred Hutchinson Canc Res Ctr, Div Clin Res, 1124 Columbia St, Seattle, WA 98104 USA

[2] Univ Washington, Sch Med, Dept Pediat, Seattle, WA 98195 USA

[3] Univ Washington, Sch Med, Dept Med, Seattle, WA 98195 USA

[4] Seattle Childrens Hosp, Hematol Oncol, Seattle, WA USA

[5] Med Coll Wisconsin, Dept Pediat, Div Hematol Oncol Blood & Marrow Transplant, 8701 Watertown Plank Rd, Milwaukee, WI 53226 USA

[6] Childrens Hosp Wisconsin, Milwaukee, WI 53201 USA

[7] Vanderbilt Univ, Pediat Hematol Oncol, 221 Kirkland Hall, Nashville, TN 37235 USA

[8] Oregon Hlth & Sci Univ, Pediat Blood & Marrow Transplant Program, Doernbecher Childrens Hosp, Portland, OR 97201 USA

来源：

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION | 2017年 / 23卷 / 10期

基金：

美国国家卫生研究院;

关键词：

Bone marrow failure; Diamond Blackfan Anemia; Shwachman-Diamond; Syndrome; Reduced-toxicity conditioning in nonmalignant diseases; SHWACHMAN-DIAMOND-SYNDROME; PAROXYSMAL-NOCTURNAL HEMOGLOBINURIA; POLYMERASE-CHAIN-REACTION; ACUTE MYELOID-LEUKEMIA; VERSUS-HOST-DISEASE; MYELODYSPLASTIC SYNDROME; NONMALIGNANT DISEASES; CONSENSUS CONFERENCE; THALASSEMIA MAJOR; GATA2; DEFICIENCY;

D O I：

10.1016/j.bbmt.2017.06.002

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Hematopoietic cell transplantation (HCT) is effective in the treatment of inherited Marrow failure disorders and other nonmalignant diseases. Conventional myeloablative conditioning regimens have been associated with high transplant-related mortality, particularly in patients with comorbid conditions. Here we report on 14 patients with marrow failure disorders (Shwachman-Diamond syndrome, n =3; Diamond Blackfan anemia, n = 4; GATA2 deficiency, n = 2; paroxysmal nocturnal hemoglobinuria, n = 4; and an undefined marrow failure disorder, n =1) who underwent HCT on a prospective, phase II, multicenter clinical trial. Patients were given HLA-matched related (n = 2) or unrelated (n=12) grafts after conditioning with treosulfan (42 g/m(2)), fludarabine (150 mg/m(2)), +/- thymoglobulin (n =11; 6 mg/kg). All patients engrafted. At a median follow-up of 3 years, 13 patients are alive with complete correction of their underlying disease. These results indicate that the combination of treosulfan, fludarabine, and thymoglobulin is effective at establishing donor engraftment with a low toxicity profile and excellent disease-free survival in patients with marrow failure disorders. (C) 2017 American Society for Blood and Marrow Transplantation.

引用

页码：1669 / 1677

页数：9

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