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Extracorporeal photopheresis (ECP) has been shown to be an effective treatment for graft-versus-host disease (GvHD). However, information regarding lymphocyte collection for ECP in children is limited. The aim of this study was to analyse and compare lymphocyte collection for ECP in children using different devices and protocols. Moreover, we have studied both safety and variables of the infused product related to treatment efficacy.This was a retrospective study of 91 patients who underwent 1524 apheresis procedures with either the COBE Spectra or Spectra Optia system. The comparison study between the Optia protocols (MNC and CMNC) was prioritized. We analysed 578 procedures using the Optia blood cell separator: 204 and 374 using the MNC and the CMNC protocol, respectively.The Optia CMNC protocol showed better collection efficiency, with increased lymphocyte collection per kg of body weight (p 0.001). On multivariate analysis, the type of protocol showed no relationship with haematocrit or platelet loss. Most procedures were well-tolerated, with the most frequent adverse events related to venous access (21.7%). Seventy-one percent of patients had either partial or complete clinical GvHD response. In the multivariate model, only two variables were associated with a better response to ECP, younger age and a greater increase of B lymphocytes after treatment.Lymphocyte collection for ECP is well-tolerated in most children, achieving complete or partial response in more than half of GvHD patients. CMNC is the optimal software to perform lymphocyte collection in children.

Introduction: In recent years, there have been changes in the management of patients with primary immune thrombocytopenia. In this study, a review is presented of the characteristics and outcomes of children with primary immune thrombocytopenia in a children’s hospital (Hospital Infantil Niño Jesús). Moreover, an analysis is made of the changes in the care of these patients diagnosed before and after 2011, when new guidelines were published by the Spanish Society of Paediatric Haematology Oncology (SEHOP). Material and methods: Data from a cohort of primary immune thrombocytopenia patients followed up in this hospital have been retrospectively reviewed. The statistical package used for the analysis was SPSS Statistics 22.0 (IBM Corp., Chicago, IL, USA). Results: A review is presented on the clinical data from 235 paediatric patients diagnosed with primary immune thrombocytopenia. It was observed that some features at diagnosis, such as age younger than five years and a previous history of infection, influenced the probability of cure. Regarding the changes in the management of patients since 2011, the steroid doses received during the first month and the first year, and the number of days corresponding to the patient’s first admission have both significantly decreased. Splenectomies were also significantly reduced. Conclusions: Since 2011, there have been changes in the medical care of our primary immune thrombocytopenia patients: they receive lower doses of steroids, they stay fewer days in the hospital, and the number of splenectomies has decreased without increasing bleeding or worsening the clinical evolution. Furthermore, it was observed that age younger than 5 years and a history of infection prior to diagnosis were related to higher chances of recovery. Resumen: Introducción: En los últimos años se han experimentado cambios en el manejo de los pacientes con trombocitopenia inmune primaria. En este estudio se revisan las características de los pacientes con trombocitopenia inmune primaria del Hospital Infantil Universitario Niño Jesús y su evolución. Además, analizamos los cambios en el abordaje de los pacientes diagnosticados antes y después del 2011, año en el que se publicó la guía de la Sociedad Española de Pediatría. Material y métodos: Se han revisado retrospectivamente los datos de pacientes con trombocitopenia inmune primaria en seguimiento en nuestro hospital desde el año 2000. El paquete estadístico utilizado para el análisis fue SPSS Statistics 22.0 (IBM Corp, Chicago, IL, EE.UU.). Resultados: Se han revisado 235 pacientes pediátricos con trombocitopenia inmune primaria observando que algunas características al diagnóstico, como la edad menor de 5 años y los antecedentes previos de infección, pueden influir en la probabilidad de recuperación. Con respecto al cambio de manejo de los pacientes, a partir de 2011 las dosis de esteroides recibidas durante el primer mes y el primer año se han reducido de forma significativa, así como el número de días del primer ingreso pasando de 5 a 3 días. Las esplenectomías también se han reducido significativamente. Conclusiones: Desde el año 2011 se han producido cambios en el abordaje de nuestros pacientes: reciben una menor dosis de esteroides, permanecen menos días ingresados y se ha reducido el número de esplenectomías sin aumentar los sangrados y sin disminuir la tasa de respuestas. Además, observamos que la edad menor de 5 años y el antecedente de infección previa al diagnóstico están relacionados con una mayor tasa de recuperación.

Introduction: Autoimmune hemolytic anemia (AIHA) is a rare and generally self-limiting disease in children. Material and methods: A descriptive cross-sectional study was performed in children under 18 years diagnosed with AIHA from January 1997 to July 2019. Clinical variables were collected and AIHA was classified according to the direct antiglobulin test (DAT) in warm AIHA (IgG+/-C3d) and cold AIHA (C3d). Response to treatment and evolution were analyzed. Results: 25 patients were included and 72% were males. The median age at diagnosis was 2 years (range 0.4–9). Fever (72%), pallor (68%), jaundice (64%), hepatosplenomegaly and coluria (48%) were the most common presenting symptoms. The median hemoglobin at diagnosis was 5.4 g/dl. DAT was positive in 96%, with detection of IgG antibodies in 76%. A single case presented negative DAT. 20% of the patients associated another cytopenia, one of which was subsequently diagnosed with common variable immunodeficiency. Concomitant viral infection was suspected or documented in 32%. Most of the cases were self-limiting and responded to corticosteroid treatment (72%). Those with partial response (24%), mainly those associated with other cytopenias, required other lines of treatment (rituximab, mycophenolate, immunoglobulins,…). Complications (32%) and relapses (26%) were detected only in warm AIHA. Conclusions: Our case series confirms that AIHA is a very rare disease in childhood. Most cases evolve favorably, although up to a quarter of them require second lines of treatment and, in exceptional cases, they need very aggressive treatments. These latter cases generally correspond to patients who present more than one cytopenia in the course of the disease. Resumen: Introducción: La anemia hemolítica autoinmune (AHAI) es una enfermedad rara en niños, generalmente autolimitada. Material y métodos: Estudio descriptivo transversal en menores de 18 años diagnosticados de AHAI desde enero 1997 a julio 2019. Se recogieron variables clínicas y se clasificaron según el test de Coombs directo (TCD) en AHAI por anticuerpos calientes (IgG+/-C3d) y fríos (C3d). Se analizó la respuesta al tratamiento y su evolución. Resultados: Se incluyeron 25 pacientes, siendo el 72% varones. La mediada de edad al diagnóstico fue 2 años (rango 0,4–9). Los síntomas predominantes fueron fiebre (72%), palidez (68%), ictericia (64%), hepatoesplenomegalia y coluria (48%). La mediana de hemoglobina al diagnóstico fue 5,4 gr/dl. En el 96% el TCD fue positivo, con detección de anticuerpos IgG en el 76%. Un solo caso presentó TCD negativo. Un 20% asociaron otra citopenia, uno de ellos fue diagnosticado posteriormente de inmunodeficiencia variable común. En un 32% se sospechó o documentó una infección viral concomitante. La mayoría de los casos fueron autolimitados y respondieron a tratamiento con corticoides (72%). Aquellos con respuesta parcial (24%), principalmente los que asociaban otras citopenias, precisaron otras líneas de tratamiento (rituximab, micofenolato, inmunoglobulinas,…). Se detectaron complicaciones (32%) y recaídas (26%) únicamente en AHAI por anticuerpos calientes. Conclusiones: Nuestra serie confirma que la AHAI es una enfermedad muy poco frecuente en la infancia. La mayoría de los casos evolucionan favorablemente, aunque hasta una cuarta parte precisan segundas líneas de tratamiento y casos excepcionales tratamientos muy agresivos. Estos últimos suelen corresponder a pacientes con más de una citopenia en la evolución de la enfermedad.

Mobilization regimes in pediatric patients at high risk for poor mobilization are not standardized across different institutions. We present a retrospective analysis of our experience with a high-dose granulocyte colony-stimulating factor (G-CSF) regime of 12 μg/Kg per body weight (BW) twice a day for 4 days used in high-risk patients.We report the results of all pediatric patients mobilized with high-dose G-CSF between January 1999 and February 2021 in our center. A successful mobilization was defined as a peripheral blood (PB) CD34Of the 262 patients included in the analysis, mobilization failure was found in 27 (10.3%). In a univariate analysis, this was associated with age, weight, baseline diagnosis, and having undergone a previous mobilization cycle, the latter being the only factor that remained significantly associated in a multivariate analysis (P = 0.03). The 54 patients (20.6%) did not reach the minimum required CD34Although most high-risk pediatric patients are successfully mobilized with the high-dose G-CSF regime, this approach does not salvage all of them and significantly increases the presence of AEs in comparison to standard-dose regimes.

Introduction: Symptomatic venous thromboembolism (VTE) is diagnosed in 3%14% of patients during pediatric acute lymphoblastic leukemia (ALL) therapy. There are well-known risk factors, but the role of others as inherited thrombophilia is still controversial. Prophylaxis with low molecular weight heparin (LMWH) has been described, but its use is not globally accepted. Methods: A retrospective multicentric study in ALL patients 1-18 years old following SEHOP-PETHEMA-2013 treatment guideline was performed to evaluate VTE rate, anticoagulant treatment, outcome, risk factors, and safety and usefulness of LMWH administration as primary thromboprophylaxis in children with inherited thrombophilia. Results: A total of 652 patients were included in the study. VTE incidence was 8.7%. Most of the cases occurred during induction therapy associated with central venous catheter. Univariant analysis showed that family history of thrombosis, presence of mediastinal mass, high-risk treatment group, and inherited thrombophilia were statistically significant risk factors. LMWH administration seemed to decrease VTE rate in patients with inherited thrombophilia and those with T-cell ALL phenotype. Conclusion: Most of the VTE cases occurred in patients without inherited thrombophilia, but when it is present, the VTE risk is higher. LMWH administration was useful to decrease VTE in these patients.

Risk factors and outcomes of GF after TCD haploidentical transplantation in children with hematological malignancies were analyzed. 148 TCD transplants were included. 78 patients were diagnosed of ALL and 70 patients of AML. 22 out of 148 patients developed GF. MVA showed that patient9 years (HR: 5.0; 95% CI: 1.1-23.0

Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for bone marrow failure or hematopoietic malignant diseases for Fanconi anemia (FA) patients. Although results have improved over the last decades, reaching more than 90% survival when a human leukocyte antigen (HLA)-identical donor is available, alternative HCT donors are still less reported. We compared HCT outcomes using HLA-mismatched unrelated donors (MMUD; n = 123) or haplo-identical donors (HDs), either using only in vivo T cell depletion (n = 33) or T cells depleted in vivo with some type of graft manipulation ex vivo (n = 59) performed for FA between 2000 and 2018. Overall survival (OS) by 24 months was 62% (53–71%) for MMUD, versus 80% (66–95%) for HDs with only in vivo T cell depletion and 60% (47–73%) for HDs with in vivo and ex vivo T cell depletion (p =.22). Event-free survival (EFS) was better for HD-transplanted FA patients with only in vivo T cell depletion 86% (73–99%) than for those transplanted from a MMUD 58% (48–68%) or those with graft manipulation 56% (42–69%) (p =.046). Grade II-IV acute graft-versus-host disease (GVHD) was 41% (MMUD) versus 40% (HDs with no graft manipulation) versus 17% (HDs with T cell depleted graft), (p =.005). No differences were found for the other transplant related outcomes. These data suggest that HDs might be considered as an alternative option for FA patients with better EFS using unmanipulated grafts. © 2021 Wiley Periodicals LLC., The authors are particularly thankful to all centers from the Severe Aplastic Anemia Working Party and the Pediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation, who kindly agreed to participate in this study (see Appendix S1). We would also like to thank Anne E. Lippinkhof, Study Coordinator, Severe Aplastic Anemia Working Party for her invaluable help.

In order to propose risk-adapted mobilization algorithms, several authors have tried to look for predictive factors of the CD34We report the results of 46 mobilizations and apheresis procedures performed in our center with unfavorable D/R ratio. Mobilization was attempted by the standard regime of G-CSF (10 mcg/kg/24 hours) in 28 cases (60.9%), with high dose G-CSF (10 mcg/kg/12 hours) in 9 cases (19.6%), and with plerixafor and G-CSF single dose regime in 9 cases (19.6%).CD34Tailoring the mobilization regime we can reach the target cell dose, even in those cases with the worst D/R ratio.