Allogeneic hematopoietic stem cell transplantation for Chediak–Higashi syndrome

The clinical outcome of allogeneic HSCT was retrospectively analyzed in eight patients with CHS. In total, six of these eight patients are alive. Four of five patients transplanted with MAC achieved prompt engraftment, and three of the four patients, including two patients with AP before transplant, are alive without disease. In contrast, three patients transplanted with RIC without active AP disease achieved prompt engraftment and survive long term. RIC‐HSCT might be an alternative treatment for CHS similar to other types of HLH, at least for patients without active AP disease.

[1]  S. Davies,et al.  Reduced-intensity conditioning hematopoietic cell transplantation is an effective treatment for patients with SLAM-associated protein deficiency/X-linked lymphoproliferative disease type 1. , 2014, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[2]  H. Kanegane,et al.  Clinical characteristics and outcomes of chédiak–Higashi syndrome: A nationwide survey of Japan , 2013, Pediatric blood & cancer.

[3]  T. Okamura,et al.  Reduced‐intensity conditioning in unrelated donor cord blood transplantation for familial hemophagocytic lymphohistiocytosis , 2012, American journal of hematology.

[4]  R. Hanada,et al.  Rituximab and cyclosporine therapy for accelerated phase Chediak–Higashi syndrome , 2011, Pediatric blood & cancer.

[5]  A. Filipovich,et al.  Reduced‐intensity conditioning haematopoietic cell transplantation for haemophagocytic lymphohistiocytosis: an important step forward , 2011, British journal of haematology.

[6]  B. Sandmaier,et al.  Defining the intensity of conditioning regimens: working definitions. , 2009, Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation.

[7]  H. Goo,et al.  A spectrum of neuroradiological findings in children with haemophagocytic lymphohistiocytosis , 2007, Pediatric Radiology.

[8]  J. Kurtzberg,et al.  Hematopoietic cell transplantation for Chediak–Higashi syndrome , 2007, Bone Marrow Transplantation.

[9]  P. Veys,et al.  Stem cell transplantation with reduced-intensity conditioning for hemophagocytic lymphohistiocytosis. , 2006, Blood.

[10]  A. Fischer,et al.  Progressive neurologic dysfunctions 20 years after allogeneic bone marrow transplantation for Chediak-Higashi syndrome. , 2005, Blood.

[11]  R. Spritz.  Genetic Defects in Chediak–Higashi Syndrome and the beige Mouse , 1998, Journal of Clinical Immunology.

[12]  A. Tichelli,et al.  Nonmalignant late effects after allogeneic stem cell transplantation. , 2003, Blood.

[13]  N. Arai,et al.  Split chimerism after allogeneic bone marrow transplantation in Chediak–Higashi syndrome , 2003, Bone Marrow Transplantation.

[14]  Benjamin C. P. Lee,et al.  Accelerated phase of Chédiak-Higashi syndrome diffuse white-matter-enhancing lesions , 1999, Pediatric Radiology.

[15]  P. Bork,et al.  Identification and mutation analysis of the complete gene for Chediak–Higashi syndrome , 1996, Nature Genetics.

[16]  E D Thomas,et al.  1994 Consensus Conference on Acute GVHD Grading. , 1995, Bone marrow transplantation.

[17]  A. Fischer,et al.  Treatment of Chediak-Higashi syndrome by allogenic bone marrow transplantation: report of 10 cases. , 1995, Blood.

[18]  K. Sullivan,et al.  Chronic graft-versus-host syndrome in man. A long-term clinicopathologic study of 20 Seattle patients. , 1980, The American journal of medicine.