Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report

被引：89

作者：

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

Petrovic, Aleksandra ^{[1
,2
]}

Brazauskas, Ruta ^{[3
]}

Liu, Xuerong ^{[3
]}

Griffith, Linda M. ^{[4
]}

Ochs, Hans D. ^{[2
]}

Bleesing, Jack J. ^{[5
]}

Edwards, Stephanie ^{[5
]}

Dvorak, Christopher C. ^{[6
]}

Chaudhury, Sonali ^{[7
]}

Prockop, Susan E. ^{[8
]}

Quinones, Ralph ^{[9
]}

Goldman, Frederick D. ^{[10
]}

Quigg, Troy C. ^{[11
]}

Chandrakasan, Shanmuganathan ^{[12
]}

Smith, Angela R. ^{[13
]}

Parikh, Suhag ^{[14
]}

Saldana, Blachy J. Davila ^{[15
]}

Thakar, Monica S. ^{[16
]}

Phelan, Rachel ^{[16
]}

Shenoy, Shalini ^{[17
]}

Forbes, Lisa R. ^{[18
]}

Martinez, Caridad ^{[19
]}

Chellapandian, Deepak ^{[20
]}

Shereck, Evan ^{[21
]}

Miller, Holly K. ^{[22
]}

Kapoor, Neena ^{[23
]}

Barnum, Jessie L. ^{[24
]}

Chong, Hey ^{[24
]}

Shyr, David C. ^{[25
]}

Chen, Karin ^{[26
]}

Abu-Arja, Rolla ^{[27
]}

Shah, Ami J. ^{[28
]}

Weinacht, Katja G. ^{[28
]}

Moore, Theodore B. ^{[29
]}

Joshi, Avni ^{[30
]}

DeSantes, Kenneth B. ^{[31
]}

Gillio, Alfred P. ^{[32
]}

Cuvelier, Geoffrey D. E. ^{[33
]}

Keller, Michael D. ^{[34
,35
]}

Rozmus, Jacob ^{[36
]}

Torgerson, Troy ^{[2
]}

Pulsipher, Michael A. ^{[23
]}

Haddad, Elie ^{[37
]}

Sullivan, Kathleen E. ^{[38
]}

Logan, Brent R. ^{[3
]}

Kohn, Donald B. ^{[29
]}

Puck, Jennifer M. ^{[6
]}

Notarangelo, Luigi D. ^{[39
]}

Pai, Sung-Yun ^{[40
,41
]}

机构：

[1] Fred Hutchinson Canc Res Ctr, 1100 Fairview Ave North,Suite D5-289,POB 19024, Seattle, WA 98109 USA

[2] Univ Washington, Dept Pediat, Seattle Childrens Hosp, Seattle, WA 98195 USA

[3] Med Coll Wisconsin, Div Biostat, Milwaukee, WI 53226 USA

[4] NIAID, Div Allergy Immunol & Transplantat, NIH, 9000 Rockville Pike, Bethesda, MD 20892 USA

[5] Univ Cincinnati, Dept Pediat, Div Bone Marrow Transplantat & Immune Deficiency, Cincinnati Childrens Hosp Med Ctr, Cincinnati, OH USA

[6] Univ Calif San Francisco, Benioff Childrens Hosp, Pediat Allergy Immunol & Blood & Marrow Transplan, San Francisco, CA 94143 USA

[7] Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Feinberg Sch Med, Div Hematol Oncol & Stem Cell Transplantat, Chicago, IL 60611 USA

[8] Mem Sloan Kettering Canc Ctr, Dept Pediat, Bone Marrow Transplant Serv, New York, NY 10021 USA

[9] Univ Colorado, Dept Pediat, Pediat Bone Marrow Transplant BMT & Cellular Ther, Sch Med, Aurora, CO USA

[10] Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USA

[11] Methodist Childrens Hosp, Texas Transplant Inst, San Antonio, TX USA

[12] Emory Childrens Healthcare Atlanta, Aflac Canc & Blood Disorders Ctr, Atlanta, GA USA

[13] Univ Minnesota, Div Pediat Blood & Marrow Transplantat, Minneapolis, MN USA

[14] Duke Univ, Med Ctr, Durham, NC USA

[15] George Washington Univ, Sch Med & Hlth Sci, Childrens Natl Hosp, Div Blood & Marrow Transplantat, Washington, DC 20052 USA

[16] Med Coll Wisconsin, Ctr Blood & Marrow Transplant Res, Div Pediat Hematol Oncol & Blood & Marrow Transpl, Milwaukee, WI 53226 USA

[17] Washington Univ, Sch Med, Dept Pediat, St Louis, MO 63110 USA

[18] Texas Childrens Hosp, Baylor Coll Med, Dept Pediat, Sect Immunol Allergy & Retrovirol, Baylor, TX USA

[19] Texas Childrens Hosp, Baylor Coll Med, Ctr Cell & Gene Therapy, Canc Ctr, Baylor, TX USA

[20] Johns Hopkins All Childrens Hosp, Blood & Marrow Transplant, St Petersburg, FL USA

[21] Oregon Hlth & Sci Univ, Div Pediat Hematol Oncol, Portland, OR 97201 USA

[22] Phoenix Childrens Hosp, Phoenix, AZ USA

[23] Univ Southern Calif, Transplantat & Cellular Therapy Program, Canc & Blood Dis Inst, Childrens Hosp Los Angeles,Keck Sch Med, Los Angeles, CA 90007 USA

[24] Childrens Hosp Pittsburgh, UPMC, Pittsburgh, PA 15213 USA

[25] Univ Utah, Sch Med, Div Pediat Hematol Oncol, Primary Childrens Hosp, Salt Lake City, UT USA

[26] Univ Utah, Sch Med, Dept Pediat, Div Allergy & Immunol, Salt Lake City, UT USA

[27] Nationwide Childrens Hosp, Columbus, OH USA

[28] Stanford Univ, Stanford Sch Med Pediat Stem Cell Transplantat, Dept Pediat, Div Stem Cell Transplantat & Regenerat Med, Stanford, CA 94305 USA

[29] Univ Calif Los Angeles, David Geffen Sch Med, Dept Pediat, Los Angeles, CA 90095 USA

[30] Mayo Clin, Childrens Ctr, Rochester, MN USA

[31] Univ Wisconsin, Amer Family Childrens Hosp, Madison, WI USA

[32] Hackensack Univ, Inst Pediat Canc & Blood Disorders, Med Ctr, Hackensack, NJ USA

[33] Univ Manitoba, Canc Care Manitoba, Winnipeg, MB, Canada

[34] Childrens Natl Hosp, Div Allergy & Immunol, Washington, DC USA

[35] George Washington Univ, GW Canc Ctr, Washington, DC USA

[36] Childrens & Womens Hlth Ctr British Columbia, Vancouver, BC, Canada

[37] Univ Montreal, Dept Pediat, Pediat Immunol & Rheumatol Div, CHU St Justine, Montreal, PQ, Canada

[38] Childrens Hosp Philadelphia, Allergy & Immunol, Philadelphia, PA 19104 USA

[39] NIAID, Lab Clin Immunol & Microbiol, NIH, 9000 Rockville Pike, Bethesda, MD 20892 USA

[40] Boston Childrens Hosp, Div Hematol Oncol, Boston, MA USA

[41] Dana Farber Canc Inst, Dept Pediat Oncol, Boston, MA 02115 USA

来源：

BLOOD | 2020年 / 135卷 / 23期

基金：

美国国家卫生研究院;

关键词：

BONE-MARROW-TRANSPLANTATION; POSTTRANSPLANT CYCLOPHOSPHAMIDE; SYNDROME PROTEIN; GENE-THERAPY; MUTATIONS; CHILDREN; BLOOD; IMMUNODEFICIENCY; THROMBOCYTOPENIA; WASP;

D O I：

10.1182/blood.2019002939

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Wiskott-Aldrich syndrome (WAS) is an X-linked disease caused by mutations in the WAS gene, leading to thrombocytopenia, eczema, recurrent infections, autoimmune disease, and malignancy. Hematopoietic cell transplantation (HCT) is the primary curative approach, with the goal of correcting the underlying immunodeficiency and thrombocytopenia. HCT outcomes have improved over time, particularly for patients with HLA-matched sibling and unrelated donors. We report the outcomes of 129 patients with WAS who underwent HCT at 29 Primary Immune Deficiency Treatment Consortium centers from 2005 through 2015. Median age at HCT was 1.2 years. Most patients (65%) received myeloablative busulfanbased conditioning. With a median follow-up of 4.5 years, the 5-year overall survival (OS) was 91%. Superior 5-year OS was observed in patients <5 vs >= 5 years of age at the time of HCT (94% vs 66%; overall P = .0008). OS was excellent regardless of donor type, even in cord blood recipients (90%). Conditioning intensity did not affect OS, but was associated with donor T-cell and myeloid engraftment after HCT. Specifically, patients who received fludarabine/melphalan-based reduced-intensity regimens were more likely to have donor myeloid chimerism <50% early after HCT. In addition, higher platelet counts were observed among recipients who achieved full (>95%) vs low-level (5%-49%) donor myeloid engraftment. In summary, HCT outcomes for WAS have improved since 2005, compared with prior reports. HCT at a younger age continues to be associated with superior outcomes supporting the recommendation for early HCT. High-level donor myeloid engraftment is important for platelet reconstitution after either myeloablative or busulfan-containing reduced intensity conditioning.

引用

页码：2094 / 2105

页数：12

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