title: Allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiencies: Hospital Israelita Albert Einstein experience.
abstract: OBJECTIVE
  To report the experience of a tertiary care hospital with allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiencies.
  
  
  METHODS
  Seven pediatric patients with primary immunodeficiencies (severe combined immunodeficiency: n = 2; combined immunodeficiency: n = 1; chronic granulomatous disease: n = 1; hyper-IgM syndrome: n = 2; and IPEX syndrome: n = 1) who underwent eight hematopoietic stem cell transplants in a single center, from 2007 to 2010, were studied.
  
  
  RESULTS
  Two patients received transplants from HLA-identical siblings; the other six transplants were done with unrelated donors (bone marrow: n = 1; cord blood: n = 5). All patients had pre-existing infections before hematopoietic stem cell transplants. One patient received only anti-thymocyte globulin prior to transplant, three transplants were done with reduced intensity conditioning regimens and four transplants were done after myeloablative therapy. Two patients were not evaluated for engraftment due to early death. Three patients engrafted, two had primary graft failure and one received a second transplant with posterior engraftment. Two patients died of regimen related toxicity (hepatic sinusoidal obstruction syndrome); one patient died of progressive respiratory failure due to Parainfluenza infection present prior to transplant. Four patients are alive and well from 60 days to 14 months after transplant.
  
  
  CONCLUSIONS
  Patients' status prior to transplant is the most important risk factor on the outcome of hematopoietic stem cell transplants in the treatment of these diseases. Early diagnosis and the possibility of a faster referral of these patients for treatment in reference centers may substantially improve their survival and quality of life.
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Juliana Folloni
authors.initials: JF
authors.lastname: Fernandes
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Fabio Rodrigues
authors.initials: FR
authors.lastname: Kerbauy
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Andreza Alice Feitosa
authors.initials: AA
authors.lastname: Ribeiro
authors.affiliation: Blood Bank, Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Jose Mauro
authors.initials: JM
authors.lastname: Kutner
authors.affiliation: Clinical Trials Center, Instituto Israelita de Ensino e Pesquisa Albert Eisntein - IIEPAE, São Paulo, SP, BR.
authors.forename: Luis Fernando Aranha
authors.initials: LF
authors.lastname: Camargo
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Adalberto
authors.initials: A
authors.lastname: Stape
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Eduardo Juan
authors.initials: EJ
authors.lastname: Troster
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Gabriele
authors.initials: G
authors.lastname: Zamperlini-Netto
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Alessandra Milani Prandini de
authors.initials: AM
authors.lastname: Azambuja
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Bruna
authors.initials: B
authors.lastname: Carvalho
authors.affiliation: Children's Institute, Hospital da Clínicas of Medial College, Unversidade de São Paulo - USP, São Paulo, SP, BR.
authors.forename: Mayra de Barros
authors.initials: Mde B
authors.lastname: Dorna
authors.affiliation: Medical Science College, Universidade Estadual de Campinas - UNICAMP, São Paulo, SP, BR.
authors.forename: Marluce Dos Santos
authors.initials: Mdos S
authors.lastname: Vilela
authors.affiliation: Children's Institute, Hospital da Clínicas of Medial College, Unversidade de São Paulo - USP, São Paulo, SP, BR.
authors.forename: Cristina Miuki Abe
authors.initials: CM
authors.lastname: Jacob
authors.affiliation: department of Pediatrics, Universidade Federal de São Paulo - UNIFESP, São Paulo, SP, BR.
authors.forename: Beatriz Tavares
authors.initials: BT
authors.lastname: Costa-Carvalho
authors.affiliation: Universidade Federal do Rio de Janeiro - UFRJ, Rio de Janeiro, RJ, BR.
authors.forename: Jose Marcos
authors.initials: JM
authors.lastname: Cunha
authors.affiliation: Children's Institute, Hospital da Clínicas of Medial College, Unversidade de São Paulo - USP, São Paulo, SP, BR.
authors.forename: Magda Maria
authors.initials: MM
authors.lastname: Carneiro-Sampaio
authors.affiliation: Hospital Israelita Albert Einstein - HIAE, São Paulo, SP, BR.
authors.forename: Nelson
authors.initials: N
authors.lastname: Hamerschlak