OBJECTIVES: Leukocyte adhesion deficiency is a rare primary immune disorder caused by defects of the CD18 beta-integrin molecule on immune cells. The condition usually presents in early infancy and is characterized by deep tissue infections, leukocytosis with impaired formation of pus, and delayed wound healing. Allogeneic hematopoietic stem-cell transplantation offers the possibility of curative therapy, and with patient numbers at any individual center being limited, we surveyed the transplant experience at 14 centers worldwide. METHODS: The course of 36 children with a confirmed diagnosis of leukocyte adhesion deficiency who underwent hematopoietic stem-cell transplantation between 1993 and 2007 was retrospectively analyzed. Data were collected by the registries of the European Society for Immunodeficiencies/European Group for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research. RESULTS: At a median follow-up of 62 months (extending to 14 years), the overall survival rate was 75%. Myeloablative conditioning regimens were used in 28 patients, and reduced-intensity conditioning in 8 patients, with no deaths in this subgroup. Survival rates after matched family donor and unrelated donor transplants were similar, with 11 of 14 matched family donor and 12 of 14 unrelated donor recipients alive; mortality was greatest after haploidentical transplants, after which 4 of 8 children did not survive. Twenty-seven transplant recipients were alive, with full donor engraftment in 17 cases, mixed multilineage chimerism in 7 patients, and mononuclear cell-restricted chimerism in an additional 3 cases. CONCLUSIONS: Hematopoietic stem-cell transplantation offers long-term benefit in leukocyte adhesion deficiency and should be considered as an early therapeutic option if a suitable HLA-matched stem-cell donation is available. Reduced-intensity conditioning was particularly safe, and mixed-donor chimerism seems sufficient to prevent significant symptoms, although careful long-term monitoring will be required for these patients.
OBJECTIVES:Leukocyte adhesion deficiency is a rare primary immune disorder caused by defects of the CD18 beta-integrin molecule on immune cells. The condition usually presents in early infancy and is characterized by deep tissue infections, leukocytosis with impaired formation of pus, and delayed wound healing. Allogeneic hematopoietic stem-cell transplantation offers the possibility of curative therapy, and with patient numbers at any individual center being limited, we surveyed the transplant experience at 14 centers worldwide. METHODS: The course of 36 children with a confirmed diagnosis of leukocyte adhesion deficiency who underwent hematopoietic stem-cell transplantation between 1993 and 2007 was retrospectively analyzed. Data were collected by the registries of the European Society for Immunodeficiencies/European Group for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research. RESULTS: At a median follow-up of 62 months (extending to 14 years), the overall survival rate was 75%. Myeloablative conditioning regimens were used in 28 patients, and reduced-intensity conditioning in 8 patients, with no deaths in this subgroup. Survival rates after matched family donor and unrelated donor transplants were similar, with 11 of 14 matched family donor and 12 of 14 unrelated donor recipients alive; mortality was greatest after haploidentical transplants, after which 4 of 8 children did not survive. Twenty-seven transplant recipients were alive, with full donor engraftment in 17 cases, mixed multilineage chimerism in 7 patients, and mononuclear cell-restricted chimerism in an additional 3 cases. CONCLUSIONS: Hematopoietic stem-cell transplantation offers long-term benefit in leukocyte adhesion deficiency and should be considered as an early therapeutic option if a suitable HLA-matched stem-cell donation is available. Reduced-intensity conditioning was particularly safe, and mixed-donor chimerism seems sufficient to prevent significant symptoms, although careful long-term monitoring will be required for these patients.
Authors: N J Farinha; M Duval; E Wagner; J Champagne; N Lapointe; S Barrette; B Tapiero; L Busque; M A Champagne Journal: Bone Marrow Transplant Date: 2002-12 Impact factor: 5.483
Authors: H Hattori; S Tsuruta; Y Horikoshi; K Amano; Y Takashima; K Sakashita; J Mimaya; M Kimura; T Yoshida Journal: Pediatr Int Date: 2001-06 Impact factor: 1.524
Authors: Corinne Antoine; Susanna Müller; Andrew Cant; Marina Cavazzana-Calvo; Paul Veys; Jaak Vossen; Anders Fasth; Carsten Heilmann; Nicolas Wulffraat; Reinhard Seger; Stéphane Blanche; Wilhelm Friedrich; Mario Abinun; Graham Davies; Robert Bredius; Ansgar Schulz; Paul Landais; Alain Fischer Journal: Lancet Date: 2003-02-15 Impact factor: 79.321
Authors: F Le Deist; S Blanche; H Keable; C Gaud; H Pham; B Descamp-Latscha; V Wahn; C Griscelli; A Fischer Journal: Blood Date: 1989-07 Impact factor: 22.113
Authors: Kate E Creevy; Thomas R Bauer; Laura M Tuschong; Lisa J Embree; Andrew M Silverstone; John D Bacher; Chris Romines; Julie Garnier; Marvin L Thomas; Lyn Colenda; Dennis D Hickstein Journal: Vet Immunol Immunopathol Date: 2003-10-15 Impact factor: 2.046
Authors: A Fischer; P H Trung; B Descamps-Latscha; B Lisowska-Grospierre; I Gerota; N Perez; C Scheinmetzler; A Durandy; J L Virelizier; C Griscelli Journal: Lancet Date: 1983-08-27 Impact factor: 79.321
Authors: Thomas R Bauer; Kate E Creevy; Yu-chen Gu; Laura M Tuschong; Robert E Donahue; Mark E Metzger; Lisa J Embree; Tanya Burkholder; John D Bacher; Chris Romines; Marvin L Thomas; Lyn Colenda; Dennis D Hickstein Journal: Blood Date: 2004-01-08 Impact factor: 22.113
Authors: C Thomas; F Le Deist; M Cavazzana-Calvo; M Benkerrou; E Haddad; S Blanche; W Hartmann; W Friedrich; A Fischer Journal: Blood Date: 1995-08-15 Impact factor: 22.113
Authors: Gregory M Cooper; Emily L Durham; James J Cray; Michael R Bykowski; Gary E DeCesare; Melissa A Smalley; Mark P Mooney; Phil G Campbell; Joseph E Losee Journal: Plast Reconstr Surg Date: 2011-01 Impact factor: 4.730
Authors: Rudolph Kirk R Schultz; Kevin Scott Baker; Jaap J Boelens; Catherine M Bollard; R Maarten Egeler; Mort Cowan; Ruth Ladenstein; Arjan Lankester; Franco Locatelli; Anita Lawitschka; John E Levine; Mignon Loh; Eneida Nemecek; Charlotte Niemeyer; Vinod K Prasad; Vanderson Rocha; Shalini Shenoy; Brigitte Strahm; Paul Veys; Donna Wall; Peter Bader; Stephan A Grupp; Michael A Pulsipher; Christina Peters Journal: Biol Blood Marrow Transplant Date: 2013-07-21 Impact factor: 5.742
Authors: Linda M Griffith; Morton J Cowan; Luigi D Notarangelo; Jennifer M Puck; Rebecca H Buckley; Fabio Candotti; Mary Ellen Conley; Thomas A Fleisher; H Bobby Gaspar; Donald B Kohn; Hans D Ochs; Richard J O'Reilly; J Douglas Rizzo; Chaim M Roifman; Trudy N Small; William T Shearer Journal: J Allergy Clin Immunol Date: 2009-12 Impact factor: 10.793