Haploidentical versus Cord Blood Transplantation in Pediatric AML. A Retrospective Outcome Analysis on Behalf of the Pediatric Subcommittee of GETH (Grupo Español de Trasplante Hematopoyético).

Haploidentical stem cell transplantation (Haplo-SCT) and cord blood transplantation (CBT) are both effective alternative treatments in patients suffering from acute myeloid leukemia (AML) and lacking a matched HLA donor. In the last years, many centers have abandoned CBT procedures mostly due to concern about poorer immune recovery compared with Haplo-SCT. We conducted a retrospective multicenter study comparing the outcomes using both alternative approaches in AML. A total of 122 transplants (86 Haplo-SCTs and 36 CBTs) from 12 Spanish centers were collected from 2007 to 2021. Median age at hematopoietic stem cell transplantation (HSCT) was 7 years (0.4-20). Thirty-nine patients (31.9%) showed positive minimal residual disease (MRD) at HSCT and a previous HSCT was performed in 37 patients (30.3%). The median infused cellularity was 14.4 × 106/kg CD34+ cells (6.0-22.07) for Haplo-SCT and 4.74 × 105/kg CD34+ cells (0.8-9.4) for CBT. Median time to neutrophil engraftment was 14 days (7-44) for Haplo-SCT and 17 days (8-29) for CBT (P = .03). The median time to platelet engraftment was 14 days (6-70) for Haplo-SCT and 43 days (10-151) for CBT (P < .001). Graft rejection was observed in 13 Haplo-SCTs (15%) and in 6 CBTs (16%). The cumulative incidence of acute graft versus host disease (GvHD) grades II-IV was 54% and 51% for Haplo-SCT and CBT, respectively (P = .50). The cumulative incidence of severe acute GvHD (grades III-IV) was 22% for Haplo-SCT and 25% for CBT (P = .90). There was a tendency to a higher risk of chronic GvHD in the Haplo-SCT group being the cumulative incidence of 30% for Haplo-SCT and 12% for CBT (P = .09). The cumulative incidence of relapse was 28% and 20% for Haplo-SCT and CBT, respectively (P = .60). We did not observe statistically significant differences in outcome measures between Haplo-SCT and CBT procedures: 5-year overall survival (OS) was 64% versus 57% (P = .50), 5-year disease-free survival (DFS) 58% versus 57% (P = .80), GvHD-free and relapse-free survival (GFRFS) 41% versus 54% (P = .30), and cumulative incidence of transplant-related mortality (TRM) 14% versus 15% (P = .80), respectively. In the multivariate analysis, MRD positivity and a disease status >CR1 at the time of HSCT were significantly associated with poorer outcomes (P < .05). In conclusion, our study supports that both haploidentical and cord blood transplantation show comparable outcomes in pediatric AML patients. We obtained comparable survival rates, although CBT showed a trend to lower rates of chronic GvHD and higher GFRFS, demonstrating that it should still be considered a valuable option, particularly for pediatric patients.

Severe Impairment of T-cell Immunity and Pulmonary GvHD Are Major Risk Factors for Nontuberculous Mycobacterial Infection After Pediatric Allogeneic Hematopoietic Stem Cell Transplantation.

Hematopoietic stem cell transplant recipients are prone to infectious complications. Infections caused by nontuberculous mycobacteria have increased in adults but literature in children is scarce. We report 6 episodes of disseminated or pulmonary nontuberculous mycobacteria infection among 5 pediatric hematopoietic stem cell transplant recipients. All but one were caused by Mycobacterium avium complex. Four patients died, 2 related to nontuberculous mycobacteria infection.

Use of Eculizumab in Pediatric Patients with High-Risk Transplantation-Associated Thrombotic Microangiopathy: Outcomes and Risk Factors Associated with Response and Survival. A Retrospective Study on Behalf of the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC).

Transplantation-associated thrombotic microangiopathy (TA-TMA) is associated with high morbidity and mortality. Although survival has improved significantly with the introduction of eculizumab, the need for improvement remains, especially in high-risk patients. This study aimed to describe the results obtained with eculizumab in a pediatric cohort with the attempt to define which risk factors could determine the response to treatment. We designed a national multicenter retrospective study of children treated with eculizumab for high-risk TA-TMA. The study cohort comprised 29 patients who had undergone a first (n = 28) or second (n = 1) allogeneic hematopoietic stem cell transplantation (HSCT) for malignant (n = 17) or nonmalignant (n = 12) disease. The median time from HSCT to TA-TMA diagnosis was 154 days (interquartile range [IQR], 103 to 263 days). Eleven patients (38%) who were initially diagnosed with low- to intermediate-risk TA-TMA progressed to high-risk TA-TMA (hrTA-TMA), within a median time of 4 days (IQR, 1 to 33 days). SC5b-9 was increased in 90% of 20 patients in whom it was measured. Renal (n = 12), pulmonary (n = 1), and intestinal (n = 1) biopsy confirmed the diagnosis in 12 of 14 patients (85%). Seventeen patients (58%) had extrarenal involvement with serositis (n = 13; 44,8%), pulmonary (n = 12; 41,4%), gastrointestinal (n = 8; 27.6%), cardiovascular (n = 7; 24.1%), or central nervous system (CNS) (n = 2; 6.9%) involvement. The median time from hrTA-TMA diagnosis to the initiation of eculizumab was 7 days (IQR, 1 to 8 days). Overall, 19 patients (65.5%) responded to eculizumab, of whom 17 (58.6%) achieved a complete response and 2 (6.9%) achieved a partial response. The remaining 10 patients (34.5%) did not show any of response. The overall response rate to eculizumab for TA-TMA was 27.59% (95% confidence interval [CI], 14.87% to 47.66%) at 1 month, 55.17% (95% CI, 38.43% to 73.48%) at 3 months, and 62.07% (95% CI, 45.10% to 79.13%) at 6 months after eculizumab initiation. In multivariate analysis, the pulmonary involvement decreased the probability of response (hazard ratio [HR], .18; P = .0298). The 1-year overall survival (OS) was 55.2% (95% CI, 35.6% to 71.0%) for the whole cohort and 83.3% (95% CI, 56.7% to 94.3%) for patients who responded to eculizumab. Pulmonary involvement (HR, 14.93; P = .0043) and CNS involvement (HR, 8.63; P = .0497) were associated with a statistically significant decrease in survival. We found that patients diagnosed with hrTA-TMA with pulmonary involvement had a poor response to eculizumab, and that patients with pulmonary and CNS involvement had significantly decreased survival. Given these results, we hypothesize that providing eculizumab therapy at an early stage of the disease before organ damage is established might significantly improve the response and, consequently, survival.

The potential impact of PCV-13, PCV-15 and PCV-20 vaccines in Colombia.

PURPOSE: To provide information about which pneumococcal vaccine could have greater coverage in Colombia. METHODS: This is a retrospective analysis of patients diagnosed with invasive pneumococcal disease (IPD) between 2015 and 2019 in Bogotá, Colombia. We compared the theoretical serotype coverage of the available anti-pneumococcal vaccines (i.e., PCV-10, PCV-10 SII, PCV-13, PCV-15, PCV-20, PCV-21, PCV24, PPSV-23) and the non-vaccine-covered serotypes stratified by age. RESULTS: 690 IPD cases were included. In children ≤5 y/o, of the approved vaccines PCV-20 showed the most theoretical protection (71.3 % [149/209]), while in adults aged 18-64 y/o was PCV-20 (61.8 % [164/265]), and in those ≥65 y/o was PPSV-23 (58.1 % [100/172]) followed by PCV-20 (55.2 % [95/172]). The non-covered serotypes represented one-third of the cohort (33.9 % [234/690]), being 6C (20.5 % [48/234]), 15A (12.8 % [30/234]), and 23A (11.5 % [27/234]) the most prevalent. CONCLUSION: Introducing PCV-20 for children and PCV-20 along with a PPSV-23 booster in adults may reduce IPD frequency in all ages in Colombia. The inclusion of non-covered serotypes is required for future vaccines.

An artificial intelligence-driven predictive model for pediatric allogeneic hematopoietic stem cell transplantation using clinical variables.

BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is a procedure with high morbidity and mortality. Identifying patients for maximum benefit and risk assessment is crucial in the decision-making process. This has led to the development of predictive risk models for HSCT in adults, which have limitations when applied to pediatric population. Our goal was to develop an automatic learning algorithm to predict survival in children with malignant disorders undergoing HSCT. METHODS: We studied allogenic HSCTs performed on children with malignant disorders at a third-level hospital between 1991 and 2021. Survival was analyzed using the Kaplan-Meier method, log-rank test for the univariate analysis, and Cox regression for the multivariate analysis. A prognostic index was constructed based on these findings. Lastly, we constructed a predictive model using a random forest algorithm to forecast 1-year survival after HSCT. RESULTS: We analyzed 229 HSCTs in 201 patients with a median follow-up of 1.64 years. Variables that impacted on the multivariate analysis were older age (hazard ratio [HR] 1.40, 95% confidence interval [CI] 1.12-1.76, p = .003), oldest period of HSCT (HR 0.46, 95% CI 0.29-0.73, p < .001), and mismatched donor (HR 2.65, 95% CI 1.51-4.65, p = .001). Our prognostic index was associated with 3-year overall survival (OS; p < .001). A random forest was developed using as variables: diagnosis, age, year of HSCT, time from diagnosis to HSCT, disease stage, donor type, and conditioning. This achieved 72% accuracy in predicting 1-year OS. CONCLUSIONS: Our index and random forest was effective in predicting 1-year survival. However, further validation in diverse populations is necessary to establish their generalizability.

Interpretación de gases arteriovenosos. Una guía práctica. Revisión de tema / Interpretation of arteriovenous gases. A practical guideline. A review

La evaluación del estado ácido base y de la oxigenación de un paciente es fundamental en cualquier escenario clínico, sea en consulta externa, en un servicio de urgencias, hospitalización, en cirugía o en la unidad de cuidado intensivo. El conocimiento de las bases fisiopatológicas es de suma importancia para el entendimiento y adecuada interpretación de estas condiciones. El objetivo de esta revisión es proveer las bases de conocimiento necesarias para el abordaje adecuado de los gases arteriovenosos y proponer un modelo para la compresión e interpretación de estos. Este artículo aborda las bases fisiopatológicas de las alteraciones ácido base, los modelos existentes en su compresión, el modelo propuesto para su abordaje diagnóstico, sus diagnósticos diferenciales, el enfoque de la hipoxemia, la interpretación de los gases arteriovenosos y las variables que se pueden obtener de estos, el enfoque de la acidosis láctica y unos ejemplos del modelo propuesto.

The evaluation of the acid-base status and the oxygenation of a patient is fundamental in any clinical setting, be it in an outpatient clinic, in an emergency service, hospitalization, in surgery or in the intensive care unit. Knowledge of the pathophysiological bases is of the utmost importance for the understanding and adequate interpretation of these conditions. The objective of this review is to provide the necessary knowledge for the adequate understanding of arteriovenous gases and to propose a model for their comprehension and interpretation. This article addresses the pathophysiological bases of acid-base disorders, the existing models in their comprehension, the model proposed for their diagnostic approach, their differential diagnoses, the diagnostic approach to hypoxemia, the interpretation of arteriovenous gases and the variables that can be obtained from them, the diagnostic approach of lactic acidosis and some examples of the proposed model.

Síndrome de Mounier Kuhn en una paciente de 78 años con fibrosis pulmonar / Mounier Kuhn syndrome in a 78-year-old patient with pulmonary fibrosis

Resumen Se presenta el caso de una mujer de 78 años de edad con fibrosis pulmonar idiopàtica, quien consultó por exacerbación de sus síntomas respiratorios, a quien se le realizan estudios radiológicos, donde se evidencia dilatación de la vía aérea, previamente no descrita, y se realiza diagnóstico de síndrome de Mounier Kuhn. Hasta donde el conocimiento alcanza, es el primer caso reportado en Colombia y la tercera persona de mayor edad reportada en el mundo. Este es un hallazgo incidental, dado que desde el punto de vista fisiopatológico no hay ninguna relación causal entre la fibrosis pulmonar idiopática y el síndrome de Mounier Kuhn. Sin embargo, su presencia puedo afectar aún más el compromiso funcional, dado el eventual colapso de la vía aérea, lo cual puede llevar a intervenciones terapéuticas adicionales, como colocación de stent. (Acta Med Colomb 2017: 42-198-201).

Abstract The case of a 78-year-old woman with idiopathic pulmonary fibrosis who consulted for exacerbation of her respiratory symptoms is presented. Radiologic studies showed dilation of the airway that was not previously described, and for this reason the diagnosis of Mounier Kuhn Syndrome was made. To our knowledge, this is the first case reported in Colombia and the third oldest person reported in the world. This is an incidental finding, since from the pathophysiological point of view there is no causal relationship between idiopathic pulmonary fibrosis and Mounier Kuhn's syndrome. However, its presence may further affect the functional compromise, given the eventual collapse of the airway, which may lead to additional therapeutic interventions, such as stent placement. (Acta Med Colomb 2017: 42-198-201).

Características reprodutivas de mulheres de 14 a 49 anos e como usam os anticoncepcionais orais / Reproductives characteristics in woman form 14 to 49 years old and how they use oral contraceptives

O trabalho descreve a pesquisa na cidade de Curitiba no sul do Brasil. Foi executada com mulheres na faixa de 14 a 49 anos, com o objetivo de verificar qual método anticoncepcional que usa normalmente, conhecimento sobre o período fértil e como o anticoncepcional oral é utilizado. Entre 100 mulheres, três métodos säo os mais utilizados: anticoncepcional oral (43 por cento) e a laqueadura tubárea (22 por cento) e o preservativo masculino (17, 1 por cento). Desconhecimento sobre o período fértil ocorreu em 88 (por cento) das mulheres entrevistadas. De 100 mulheres entrevistadas (82 por cento), tinham usado a pílula em algum momento de sua vida. Considerou-se que a forma certa de uswo destes anticoncepcionais é manter intervalo de 7 dias entre dois ciclos, reiniciando a pílula no oitavo dia de pausa, independente da data da menstruaçäo. As mulheres que já tinham usado a pílula, inventaram uma nova "Tabelinha, porque näo tem relaçä sexual no período de intervalo das embalagens das pílulas, näo tendo relaçöes com medo de engravidar