Successful hematopoietic cell transplantation in a patient with X-linked agammaglobulinemia and acute myeloid leukemia

被引:26
作者
Abu-Arja, Rolla F. [1 ]
Chernin, Leah R. [2 ]
Abusin, Ghada [3 ]
Auletta, Jeffery [1 ]
Cabral, Linda [4 ]
Egler, Rachel [4 ]
Ochs, Hans D. [5 ,6 ]
Torgerson, Troy R.
Lopez-Guisa, Jesus [5 ,6 ]
Hostoffer, Robert W. [2 ]
Tcheurekdjian, Haig [2 ]
Cooke, Kenneth R. [7 ]
机构
[1] Nationwide Childrens Hosp, Pediat Blood & Marrow Transplant Program, Columbus, OH 43205 USA
[2] Case Western Reserve Univ, Allergy Immunol Associates Inc, Cleveland, OH 44106 USA
[3] Univ Iowa, Childrens Hosp, Pediat Bone Marrow Transplant Program, Iowa City, IA USA
[4] Rainbow Babies & Childrens Hosp, Pediat Bone Marrow Transplant, Case Med Ctr, Cleveland, OH 44106 USA
[5] Univ Washington, Seattle, WA 98195 USA
[6] Seattle Childrens Res Inst, Seattle, WA USA
[7] Johns Hopkins Univ, Pediat Blood & Marrow Transplant Sidney Kimmel Ca, Baltimore, MD USA
关键词
acute myeloid leukemia; stem cell transplantation; X-linked agammaglobulinemia; GENE; THERAPY; DISEASE; BTK;
D O I
10.1002/pbc.25554
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
X-linked agammaglobulinemia (XLA) is a primary immunodeficiency characterized by marked reduction in all classes of serum immunoglobulins and the near absence of mature CD19(+) B-cells. Although malignancy has been observed in patients with XLA, we present the first reported case of acute myeloid leukemia (AML) in a patient with XLA. We also demonstrate the complete correction of the XLA phenotype following allogeneic hematopoietic cell transplantation for treatment of the patient's leukemia. Pediatr Blood Cancer 2015;62:1674-1676. (c) 2015 Wiley Periodicals, Inc.
引用
收藏
页码:1674 / 1676
页数:3
相关论文
共 21 条
[1]  
Burroughs L, 2010, CELL THER TRANSPLANT, V2, P1
[2]   Stable hematopoietic cell engraftment after low-intensity nonmyeloablative conditioning in patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome [J].
Burroughs, Lauri M. ;
Torgerson, Troy R. ;
Storb, Rainer ;
Carpenter, Paul A. ;
Rawlings, David J. ;
Sanders, Jean ;
Scharenberg, Andrew M. ;
Skoda-Smith, Suzanne ;
Englund, Janet ;
Ochs, Hans D. ;
Woolfrey, Ann E. .
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2010, 126 (05) :1000-1005
[3]   Analysis of clinical presentations of Bruton Disease: A review of 20 years of accumulated data from pediatric patients at Severance Hospital [J].
Chun, Jin-Kyong ;
Lee, Taek Jin ;
Song, Jae Woo ;
Linton, John A. ;
Kim, Dong Soo .
YONSEI MEDICAL JOURNAL, 2008, 49 (01) :28-36
[4]   Clinical findings leading to the diagnosis of X-linked agammaglobulinemia [J].
Conley, ME ;
Howard, V .
JOURNAL OF PEDIATRICS, 2002, 141 (04) :566-571
[5]   Analysis of the Bruton's tyrosine kinase gene promoter reveals critical PU1 and SP1 sites [J].
Himmelmann, A ;
Thevenin, C ;
Harrison, K ;
Kehrl, JH .
BLOOD, 1996, 87 (03) :1036-1044
[6]   Mutation screening of the BTK gene in 56 families with X-linked agammaglobulinemia (XLA):: 47 unique mutations without correlation to clinical course [J].
Holinski-Feder, E ;
Weiss, M ;
Brandau, O ;
Jedele, KB ;
Nore, B ;
Bäckesjö, CM ;
Vihinen, M ;
Hubbard, SR ;
Belohradsky, BH ;
Smith, CIE ;
Meindl, A .
PEDIATRICS, 1998, 101 (02) :276-284
[7]   X-Linked Agammaglobulinemia Associated with B-Precursor Acute Lymphoblastic Leukemia [J].
Hoshino, Akihiro ;
Okuno, Yusuke ;
Migita, Masahiro ;
Ban, Hideki ;
Yang, Xi ;
Kiyokawa, Nobutaka ;
Adachi, Yuichi ;
Kojima, Seiji ;
Ohara, Osamu ;
Kanegane, Hirokazu .
JOURNAL OF CLINICAL IMMUNOLOGY, 2015, 35 (02) :108-111
[8]   The health status and quality of life of adults with X-linked agammaglobulinemia [J].
Howard, V ;
Greene, JM ;
Pahwa, S ;
Winkelstein, JA ;
Boyle, JM ;
Kocak, M ;
Conley, ME .
CLINICAL IMMUNOLOGY, 2006, 118 (2-3) :201-208
[9]   Stem cell transplants for patients with X-linked agammaglobulinemia [J].
Howard, V ;
Myers, LA ;
Williams, DA ;
Wheeler, G ;
Turner, EV ;
Cunningham, JM ;
Conley, ME .
CLINICAL IMMUNOLOGY, 2003, 107 (02) :98-102
[10]   B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia [J].
Kerns, Hannah M. ;
Ryu, Byoung Y. ;
Stirling, Brigid V. ;
Sather, Blythe D. ;
Astrakhan, Alexander ;
Humblet-Baron, Stephanie ;
Liggitt, Denny ;
Rawlings, David J. .
BLOOD, 2010, 115 (11) :2146-2155