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We analyzed the outcomes of 26 consecutive patients with acquired severe aplastic anemia (SAA) undergoing peripheral blood stem cell transplantation (PBSCT) with partial ex vivo T cell depletion with a targeted T cell dose from HLA-identical sibling donors. The median patient age was 37 years (range, 3 to 63 years). Four patients with uncontrolled pneumonia at the time of transplantation died, on days +1, +2, +21, and +26. All evaluable patients engrafted, with a median time to neutrophil recovery of 11 days (range, 10 to 14 days) and a median time to platelet recovery of 19 days (range, 8 to 53 days). Two patients had transient grade I acute graft-versus-host disease (GVHD) with skin involvement, but no patients developed grade II-IV acute GVHD. Two patients had mild skin chronic GVHD, and 1 patient had moderate chronic GVHD with ocular involvement. No relapse was observed after a median follow-up of 114 months (range, 4 to 233 months). The overall cumulative incidence of TRM at 10 years was 19%, whereas it was 5% for those with a Karnofsky Performance Status (KPS) score >60 at the time of transplantation. Disease-free survival, overall survival, and GVHD and relapse-free survival at 10 years were 81%, 81%, and 80%, respectively, for all patients and 95%, 95%, and 90%, respectively, for patients with a KPS score >60 at transplantation. Our data indicate that PBSCT with partial ex vivo T cell-depleted targeted cell dose grafts from an HLA-identical sibling donor is a feasible, safe, and effective approach to reduce GVHD and cure patients with SAA., (Copyright © 2019 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Primary immune thrombocytopenia, formerly known as immune thrombocytopenic purpura, is a disease for which the clinical and therapeutic management has always been controversial. The ITP working group of the Spanish Society of Paediatric Haematology and Oncology has updated its guidelines for diagnosis and treatment of primary immune thrombocytopenia in children, based on current guidelines, bibliographic review, clinical assays, and member consensus. The main objective is to reduce clinical variability in diagnostic and therapeutic procedures, in order to obtain best clinical results with minimal adverse events and good quality of life., (Copyright © 2019 Asociación Española de Pediatría. Publicado por Elsevier España, S.L.U. All rights reserved.)

Introduction: Haematopoietic stem cell transplantation (HSCT) involves implanting cellular elements capable of generating a new and healthy haematopoietic system. Reduced intensity conditioning (RIC) consists of an immunosuppressive treatment to facilitate a progressive implant with lower morbidity. This type of conditioning can also lead to myelosuppression, which is potentially reversible over time. Reduced intensity conditioning enables HSCT to be performed on patients with genetic diseases for whom added comorbidity is undesirable due to the high doses of chemotherapy that accompanies conventional myeloablative regimens., Patients and Methods: An analysis was performed on the outcomes of 68 paediatric patients with genetic diseases who underwent HSCT with RIC between 2005 and 2013 in the of Paediatric Haematopoietic Stem Cell Transplantation Units that are part of the Spanish Working Group for Bone Marrow Transplantation in Children. A multicentre study was conducted including 68 patients, of whom 43 had Primary Immunodeficiency, 21 with congenital haematological diseases, and 4 with metabolic diseases., Results: Fifty (73.5%) of the 68 patients were still alive. The Overall Survival (OS) at nine years was 0.74. Twenty-three (33.8%) had some event during the course of the HSCT, with an event-free survival rate of 0.66. The OS in patients with haematological diseases was 0.81, being 0.7 in primary immunodeficiencies, and 0.4 in metabolic diseases. No significant difference was observed between the 3 groups of diseases. As regards the source of haematopoietic progenitors, there was an OS rate of 0.74 in patients transplanted with peripheral blood, 0.70 with bone marrow, and 0.70 and with cord blood, with no statistically significant differences., Conclusions: Favourable results have been obtained in HSCT with reduced intensity conditioning in genetic diseases. It should be noted that the risks and benefits of the RIC in patients with metabolic diseases need to be assessed on an individual basis., (Copyright © 2017 Asociación Española de Pediatría. Publicado por Elsevier España, S.L.U. All rights reserved.)

Primary immune thrombocytopenia (ITP), formerly known as immune thrombocytopenic purpura, is a disease in which clinical and therapeutic management has always been controversial. The ITP working group of the Spanish Society of Paediatric Haematology and Oncology has updated its guidelines for diagnosis and treatment of ITP in children based on current guidelines, literature review, clinical trials and member consensus. The primary objective was to lessen clinical variability in diagnostic and therapeutic procedures in order to obtain best clinical results with minimal adverse events and good quality of life., (Copyright © 2010 Asociación Española de Pediatría. Published by Elsevier Espana. All rights reserved.)

A major problem in the treatment of haemophilia A is the development of inhibitors (antibodies) against factor VIII. We report the case of a newborn male with no family history of haemophilia who developed an intracerebral haemorrhage. On day 10 post-delivery severe haemophilia A was diagnosed and treatment with recombinant FVIII (rFVIII) concentrate was started. Seventy-two hours later the presence of inhibitors was suspected because high doses of rFVIII were required to maintain therapeutic FVIII plasma levels. Days after, the inhibitor was detected. The quick detection of the inhibitor in this newborn haemophiliac allowed us to start the immunotolerance early, without interruption in the administration of rFVIII.

Thirty-six haemophilia A, HIV-negative, previously treated patients were changed therapy to a highpurity and double-inactivated (solvent/detergent and dry-heating) previously unused factor VIII concentrate. The mean age of these patients was 27 years at the time of the change. Twenty-three patients were severe haemophiliacs (FVIII:C < 0.02 IU mL-1), seven moderate (FVIII:C between 0.02 and 0.05 IU mL-1) and six mild (FVIII:C > 0.05 IU mL-1). The mean follow-up with this single product was 16 months, with 82 accumulated exposure days and the mean consumption was 117,300 IU of FVIII corresponding to a mean of six batches per patient. No patient developed FVIII inhibitors (upper limit of the CI95: 7.98%), resulting in an incidence rate of 0/48 patient-years (upper limit of the CI95: 77/1000 patient-years). The change in therapy to this new factor VIII concentrate was not associated with the appearance of inhibitors.

Arbitrarily primed polymerase chain reaction (AP-PCR) was used to differentiate strains of Legionella pneumophila isolated from different water sources in a resort hotel in Benidorm, Alicante, Spain, where an outbreak of Legionnaires' disease occurred among a group of tourists between 65 and 80 years of age. All isolates were L. pneumophila serogroup 1, subtype Pontiac (Knoxville 1). Five different patterns (P1 to P5) were obtained by AP-PCR. The number of bands per pattern varied between 4 and 11. Patterns P1 and P2 represented 60 and 20% of L. pneumophila isolates, respectively. Since different subpopulations of L. pneumophila coexisted (up to three different AP-PCR patterns were identified in a single room), it was not possible to link an individual L. pneumophila strain to the occurrence of this outbreak.

Purpose: To assess the incidence of AIDS, along with its origin and mortality rate, in a group of sero-positive haemophiliacs with long follow-up., Material and Methods: The progression into AIDS in 94 HIV-seropositive haemophiliacs (88 with haemophilia A and 6 with haemophilia B) followed since 1986 has been analyzed. The mean age was 19 years and 64% of the patients had severe haemophilia. All of them had been previously treated with non virus-inactivated factor concentrates. Was detected HIV antigen in 13 of them and 32 were treated with zidovudine. Actuarial analysis started from seroconversion date or from the first positive result and the average observation time was 90 months (5-97)., Results: Sixteen patients developed AIDS, resulting in an eight-years accumulative actuarial indice of about 24%. AIDS incidence was lower (p less than 0.0001) in haemophilia A (21%) than in haemophilia B (67%). The eight-years progression rate to AIDS showed a significant dependence on patient age, it being higher in patients aged greater than 30 (77%) than in those aged 15-30 (30%) and less than 15 (9%). Patients who presented CD4+ counts lower than 0.5 x 10(9)/L or CD4/CD8 ratios lower than 0.5 at the beginning of the observation period had a greater 8-year AIDS incidence (63% and 57% respectively) than the remaining patients (16% and 12%). At AIDS diagnosis, all patients had CD4+ lymphocyte counts lower than 0.2 x 10(9)/L, two had detectable HIV antigenaemia and six had been previously treated with zidovudine. The cause of AIDS was an opportunistic infection in all the cases namely, P. carinii pneumonia and candidiasis, but no secondary neoplasia was registered. Nineteen patients died during the follow-up, 12 because of AIDS and 7 because of other reasons unrelated to HIV-infection., Conclusion: Our results suggest that 25% of the haemophiliacs will develop AIDS eight years after seroconversion, and a decreasing incidence is not observed. Lower progression rate to AIDS in children than in adults is confirmed.

The prevalence of HIV-antibody was studied in a cohort of 156 haemophiliacs controlled since before 1986. Ninety four patients (60%) were HIV-seropositive, and all of them had been previously treated with non-heated factor concentrates. Seroprevalence was 50% by 1983. Evidence for previous negative results was only available in 19 seropositive patients and all of them seroconverted before 1985. For the 148 patients exposed to non-heat treated factor concentrates, severe haemophiliacs and those with factor consumption greater than 100 x 10(3) had the maximal rate of seropositivity (81% and 75% respectively, p less than 0.005). Fifteen patients developed AIDS (10% of the HIV-positive), 12 of which have died. HIV infection has a high prevalence and it has become the most important cause of death in our haemophiliacs.