JULIA LOPES GARCIA

Índice h a partir de 2011
2
Projetos de Pesquisa
Unidades Organizacionais
Instituto da Criança, Hospital das Clínicas, Faculdade de Medicina - Médico

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  • conferenceObject
    Outcomes and Immune Reconstitution After T-Cell Replete Haploidentical Stem Cell Transplantation With Post-Transplantation Cyclophosphamide (PTCY) for Pediatric Patients with Primary Immunodeficiencies
    (2020) FERNANDES, Juliana Folloni; NICHELE, Samantha; ARCURI, Leonardo Javier; RIBEIRO, Lisandro; NETTO, Gabriele Zamperlini; LOTH, Gisele; RODRIGUES, Ana Luiza Melo; KUWAHARA, Cilmara; KOLISKI, Adriana; GARCIA, Julia Lopes; DAUDT, Liane Esteves; SEBER, Adriana; GOMES, Alessandra Araujo; HAMERSCHLAK, Nelson; BONFIM, Carmem
  • article 33 Citação(ões) na Scopus
    Outcomes after Haploidentical Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Patients with Primary Immunodeficiency Diseases
    (2020) FERNANDES, Juliana Folloni; NICHELE, Samantha; ARCURI, Leonardo Javier; RIBEIRO, Lisandro; ZAMPERLINI-NETTO, Gabriele; LOTH, Gisele; RODRIGUES, Ana Luiza Melo; KUWAHARA, Cilmara; KOLISKI, Adriana; TRENNEPOHL, Joanna; GARCIA, Julia Lopes; DAUDT, Liane Esteves; SEBER, Adriana; GOMES, Alessandra Araujo; FASTH, Anders; PASQUINI, Ricardo; HAMERSCHLAK, Nelson; ROCHA, Vanderson; BONFIM, Carmem
    Allogeneic hematopoietic stem cell transplantation (HCT) can cure primary immunodeficiency diseases (PID). When a HLA-matched donor is not available, a haploidentical family donor may be considered. The use of T cell-replete haploidentical HCT with post-transplantation cyclophosphamide (haplo-PTCy) in children with PID has been reported in few case series. A donor is usually readily available, and haplo-PTCy can be used in urgent cases. We studied the outcomes of 73 patients with PID who underwent haplo-PTCy, including 55 patients who did so as a first transplantation and 18 who did so as a salvage transplantation after graft failure of previous HCT. The median patient age was 1.6 years. Most of the children were male (n = 54) and had active infection at the time of transplantation (n = 50); 10 children had severe organ damage. The diagnosis was severe combined immunodeficiency (SCID) in 34 patients and non-SCID in 39 (Wiskott-Aldrich syndrome; n = 14; chronic granulomatous disease, n = 10; other PID, n = 15). The median duration of follow-up of survivors was 2 years. The cumulative incidence of neutrophil recovery was 88% in the SCID group and 84% in non-SCID group and was 81% for first transplantations and 83% after a salvage graft. At 100 days, the cumulative incidence of acute GVHD grade II-IV and III-IV was 33% and 14%, respectively. The majority of patients reached 200/mu L CD4(+) and 1000/mu L CD3(+) cell counts between 3 and 6 months. The estimated 2-year overall survival was 66%; it was 64% for SCID patients and 65% for non-SCID patients and 63% for first HCT and 77% for salvage transplantations. Twenty-five patients died, most of them due to infection early after transplantation (before 100 days). In conclusion, haplo-PTCy is a feasible procedure, can cure two-thirds of children with PID, and can be used as rescue treatment for previous graft failure. (C) 2020 American Society for Transplantation and Cellular Therapy.