Hematopoietic stem cell transplantation in children with chronic granulomatous disease: the Spanish experience.

Introduction: Hematopoietic stem cell transplantation (HCT) can cure chronic granulomatous disease (CGD). However, transplant-associated morbidity or mortality may occur, and it is still controversial which patients benefit from this procedure. The aim of this retrospective study was to evaluate the outcome of pediatric patients who received HCT in one of the Spanish pediatric transplant units. Results: Thirty children with a median age of 6.9 years (range 0.6-12.7) were evaluated: 8 patients received a transplant from a sibling donor (MSD), 21 received a transplant from an unrelated donor (UD), and 1 received a haploidentical transplant. The majority of the patients received reduced-intensity conditioning regimens based on either busulfan plus fludarabine or treosulfan. Relevant post-HCT complications were as follows: i) graft failure (GF), with a global incidence of 28.26% (CI: 15.15-48.88), 11.1% in patients with MSD (1.64-56.70) and 37.08% in unrelated donors (19.33-63.17); and ii) chronic graft-versus-host disease (GVHD), with an incidence of 20.5% (8.9-43.2), 11.1% in patients with MSD (1.64-56.70) and 26.7% in unrelated donors (10.42-58.44). Post-HCT infections were usually manageable, but two episodes of pulmonary aspergillosis were diagnosed in the context of graft rejection. The 2-year OS was 77.3% (55.92-89.23). There were no statistically significant differences among donor types. Discussion: HCT in patients with CGD is a complex procedure with significant morbidity and mortality, especially in patients who receive grafts from unrelated donors. These factors need to be considered in the decision-making process and when discussing conditioning and GVHD prophylaxis.

Cost-effectiveness analysis of defibrotide in the treatment of patients with severe veno-occlusive disease/sinusoidal obstructive syndrome with multiorgan dysfunction following hematopoietic cell transplantation in Spain.

AIMS: This study evaluated cost-effectiveness of defibrotide vs best supportive care (BSC) for the treatment of hepatic veno-occlusive disease/sinusoidal obstructive syndrome (VOD/SOS) with multiorgan dysfunction (MOD) post-hematopoietic cell transplantation (HCT) in Spain. MATERIALS AND METHODS: A two-phase Markov model, comprising a 1-year acute phase with daily cycles and a lifetime long-term phase with annual cycles, was adapted to the Spanish setting. The model included a cohort of patients with severe VOD/SOS (defined as VOD/SOS with MOD) post-HCT. For the acute phase, efficacy and VOD/SOS-related length of stay were obtained from a phase 3 defibrotide study (NCT00358501). VOD/SOS-related hospital stays were 7.5 and 23.2 days in defibrotide-treated and BSC patients, respectively. Defibrotide-treated patients spent 30% of their stay in the intensive care unit vs 60% in BSC patients. Assumptions for the long-term phase and utility values were obtained from the literature. Costs were from the Spanish Health System perspective (€2019). Defibrotide cost was based on 25 mg/kg/day over 17.5 days, using local expert opinion. Life-years (LYs), quality-adjusted LYs (QALYs), and costs were estimated over a lifetime horizon, applying a 3% discount rate for costs and outcomes. Sensitivity analyses assessed the robustness of the results. RESULTS: Defibrotide produced an additional 1.214 QALYs and 1.348 LYs vs BSC, with a total cost of €33,708 more than BSC alone. However, defibrotide resulted in savings up to €16,644/patient for cost of hospital stay. Difference between costs and effective measures led to ratios of €27,757/QALY and €25,007/LY gained. Additional hospital stays had the greatest influence on base-case results. Probabilistic analysis confirmed the robustness of the deterministic results. LIMITATIONS: Limitations include use of historical controls and assumptions extrapolated from the literature. CONCLUSIONS: This cost-effectiveness model, adapted to the Spanish setting, showed that defibrotide is a cost-effective alternative to BSC alone in patients with severe VOD/SOS post-HCT.

Caso clínico: hipercalcemia refractaria asociada a linfoma b, una emergencia oncológica / Clinical case: Refractory hypercalcemia associated to B-cell lymphoma, an oncological emergency

La hipercalcemia asociada a procesos tumorales es un hallazgo poco frecuente en la edad pediátrica. El manejo terapéutico va encaminado a favorecer la calciuresis con diversos métodos farmacológicos e, incluso, técnicas de depuración extrarrenal. El objetivo de la exposición de este caso clínico es presentar a un paciente con hipercalcemia grave refractaria, que solo respondió a un tratamiento etiológico precoz con el empleo de quimioterapia, y se evitaron así las posibles complicaciones secundarias a dicha alteración electrolítica.

Hypercalcemia as a paraneoplastic syndrome is rare in children. Therapeutic management is aimed at promoting calciuresis with various pharmacological methods, even with extrarenal purification techniques. The aim of presenting this case is to highlight the importance of early etiologic treatment through chemotherapy as an urgent treatment in a refractory and severe hypercalcemia case, in order to avoid possible secondary complications due to this electrolyte disturbance.

Caso clínico: hipercalcemia refractaria asociada a linfoma b, una emergencia oncológica / Clinical case: Refractory hypercalcemia associated to B-cell lymphoma, an oncological emergency

La hipercalcemia asociada a procesos tumorales es un hallazgo poco frecuente en la edad pediátrica. El manejo terapéutico va encaminado a favorecer la calciuresis con diversos métodos farmacológicos e, incluso, técnicas de depuración extrarrenal. El objetivo de la exposición de este caso clínico es presentar a un paciente con hipercalcemia grave refractaria, que solo respondió a un tratamiento etiológico precoz con el empleo de quimioterapia, y se evitaron así las posibles complicaciones secundarias a dicha alteración electrolítica.(AU)

Hypercalcemia as a paraneoplastic syndrome is rare in children. Therapeutic management is aimed at promoting calciuresis with various pharmacological methods, even with extrarenal purification techniques. The aim of presenting this case is to highlight the importance of early etiologic treatment through chemotherapy as an urgent treatment in a refractory and severe hypercalcemia case, in order to avoid possible secondary complications due to this electrolyte disturbance.(AU)

[Clinical case: Refractory hypercalcemia associated to B-cell lymphoma, an oncological emergency]. / Caso clínico: hipercalcemia refractaria asociada a linfoma b, una emergencia oncológica.

Hypercalcemia as a paraneoplastic syndrome is rare in children. Therapeutic management is aimed at promoting calciuresis with various pharmacological methods, even with extrarenal purification techniques. The aim of presenting this case is to highlight the importance of early etiologic treatment through chemotherapy as an urgent treatment in a refractory and severe hypercalcemia case, in order to avoid possible secondary complications due to this electrolyte disturbance.

Quality of chest compressions by Down syndrome people: a pilot trial.

INTRODUCTION: Immediate bystander cardiopulmonary resuscitation (CPR) is essential for survival after out-of-hospital cardiac arrest. Down syndrome (DS) citizens have improved their active engagement in society. The objective of this pilot trial was to investigate if they are able to perform quality chest compression-only CPR after a brief training. METHODS: Nineteen DS young people (15-30 year old) and 20 University level subjects (18-29 year old) were trained by means of a short video and a brief hands-on session on manikins, to perform chest compression-only CPR. All participants were naïve in CPR. Chest compression (CC) quality (percentage of correct CC, CC rate and depth and chest complete release) was measured during a 2min test. CPR quality goal was according to 2010 European Resuscitation Council guidelines. RESULTS: DS people had similar weight, lower height and a higher BMI than controls. They were able to deliver chest compression-only CPR but with higher mean CC rate (140±30 vs 123±12CC/min, p=0.03), less mean CC depth (35.4±10.3 vs 47.2±9.6mm, p=0.03) and lower % of full correct CC (13±18 vs 39±37, p=0.02) than controls. Differences were maintained when first and second minute of test were compared. CONCLUSIONS: After a short instruction based on a brief video and hands-on session DS people were able to deliver CC but with poor quality.