Background Hemophagocytic lymphohistiocytosis (HLH) is an exaggerated inflammatory reaction secondary to a host’s inadequate immune response causing a self-perpetuating loop of altered regulation. Signs and symptoms of HLH are compatible with other common diseases and are nonspecific. Underdiagnosis makes it difficult to estimate the real incidence of HLH, especially in developing countries. Methods Retrospective, descriptive study of pediatric patients admitted to a high-complexity institution in Cali, Colombia between 2012 and 2019 with HLH diagnosis. Medical history review to complete an electronic database and a secondary, descriptive analysis was carried out. The study was approved by the Institutional Ethics Committee. Results Twenty-one patients were included. 52.4 % of the population was male with a median age of 9.3 years [IQR (3.0-13.7 years)]. More than half of patients (66.6 %) had viral disease at diagnosis, the most frequent being Epstein-Barr Virus (EBV) (52.3 %) and dengue (14.3 %). Three patients had confirmed gene mutations (G6PC3, XIAP, and UNC13D). 95 % of the patients were treated with the HLH 2004 protocol, half of them received incomplete protocol with intravenous immunoglobulin (IVIG) and/or systemic steroids, while the other half received the complete protocol including etoposide and cyclosporine. More than three-fourths (76.2 %) required admission to an ICU with a median stay of 14 days [IQR (11–37 days)] and a median hospital stay of 30 days [IQR (18–93 days)]. 14.3 % (n = 3) of patients died. Conclusions HLH is a complex disease that requires multidisciplinary management with secondary HLH due to EBV infection being a common cause. There is increasing awareness of HLH diagnosis in developing countries such as Colombia which can offer earlier treatment options and better outcomes.
Introduction: Hemophagocytic lymphohistiocytosis (HLH) is an exaggerated inflammatory reaction secondary to a host's inadequate immune response causing a self-perpetuating loop of altered regulation. Signs and symptoms of HLH are compatible with other common diseases and are nonspecific. Underdiagnosis makes it difficult to estimate the real incidence of HLH, especially in developing countries. Materials and Methods: Retrospective, descriptive study of hospitalized pediatric patients admitted to a high-complexity institution in Cali, Colombia between 2012 and 2019 with HLH diagnosis. Medical history review to complete a virtual database. A secondary descriptive analysis was carried out. The study was approved by the Institutional Ethics Committee. Results: Twenty-one patients were included. 52.4% of the population was male with a median age of 9.3 years [IQR (3.0-13.7years)]. More than half of patients (66.6%) had viral isolation at diagnosis, the most frequent being Epstein-Barr Virus (EBV) (52.3%) and dengue (14.3%). Three patients had gene mutations (LYST, XIAP, and UNC13D). Ninety-five percent of the patients were treated with the HLH 2004 protocol, half of them received incomplete protocol with IgIV at high doses and/or systemic steroids, while the other half received the complete protocol including etoposide and cyclosporine. More than three-fourths (76.2%) required admission to an ICU with a median stay of 14 days [IQR (11-37 days)] and a median hospital stay of 30 days [IQR (18-93 days)]. 14.3% (n = 3) of patients died. Conclusions: HLH is an underdiagnosed pathology that requires greater sensitization in developing countries in order to make early diagnoses and obtain better outcomes.
matched sibling bone marrow transplantation results in approximately 85% event free survival in high-risk patients with Sickle Cell Disease (SCD) (Walters, NEJM, 2006). However, this approach is limited by 5-10% transplantrelated mortality, 5-10% primary graft failure and the late effects of Bu/CY. Alternate approaches include reduced toxicity but more immunosuppressive conditioning and the use of sibling cord blood (Geyer/Cairo, BJH, 2011 and Freed/ Cairo, BMT, 2012). Objective: To determine the safety, donor chimerism and long term organ function associated with Bu 12.8-16 mg/kg, fludarabine180 mg/m 2 and alemtuzumab 54 mg/m 2 (BFA) reduced toxicity conditioning (RTC) prior to HLA-matched sibling donor transplantation in pediatric recipients with high risk SCD. Methods: Patients 21 years of age with HbSS, HbSC, HbSb + or HbSb 0 were eligible if highly symptomatic (such as 2 vaso-occlusive crises per year requiring narcotics, acute chest syndrome, stroke, retinopathy, splenic sequestration) and with an HLA-matched sibling donor. Conditioning consisted of busulfan (4mg/kg x 4d < 4 yrs and 12.8mg/ kg x 4d > 4 yrs), fludarabine (30mg/m 2 x 6d), and alemtuzumab (2mg/ m 2 x 1d, 6mg/m 2 x 2d, and 20mg/m 2 x 2d) as we have described (Styczynski/Cairo,BMT,2011). All received tacrolimus and MMF as GVHD prophylaxis as we have described (Bhatia/Cairo, BBMT, 2010). Results: 12 patients (11M:1F), median age 12 (2-19) with symptomatic SCD underwent sibling BM (n¼10) or CB (n¼2) Allogeneic Stem Cell Transplantation (AlloSCT). Median follow-up was 33 months. Median time to neutrophil and platelet engraftment for recipients of sibling BM and CB were 16 days (13-18), 18.5 days (9-43) and 39.5 days (38-41) and 74 days (73-75), respectively. The probability of grade II-IV and grade III-IV aGVHD were 16.7% and 8.3%. No patients developed cGVHD. Patients achieved mean whole-blood donor chimerism of 85, 94, 93, 93, 89 and 93% and mean erythroid (CD71) donor chimerism of 89, 89, 93, 90, 86, and 94% at days 30, 60, 100, 180, 365 and 730 post-transplant, respectively. The Kaplan-Meier probability of OS and EFS was 100% (CI 95 : 73.5-100%). Neurological, pulmonary, vascular, and splenic function were stable to improved at 2 years. Conclusions: BFA (RTC) and HLA-matched sibling bone marrow and cord blood AlloSCT in pediatric recipients results in excellent EFS, long term donor chimerism, and stable/improved organ function.
BackgroundHepatoblastoma is the most common primary malignant liver tumor in children and is usually diagnosed during the first 3 years of life. Overall survival has increased 50% due to chemotherapeutic schemes, expertise surgery centers, and liver transplantation.MethodsA retrospective collection of data was performed from pediatric patients with diagnosis of hepatoblastoma. Variables included demographic, diagnostic tools and histological classification; chemotherapy and surgical treatment; and outcomes and patient survival. The PRETEXT classification was applied, which included the risk evaluation, and according to the medical criterion in an individualized way, underwent resection or transplant. The morbidity of patients was evaluated by the Clavien-Dindo classification. Statistical analysis was performed according to the distribution of data and the survival analysis was carried out using the Kaplan-Meier method.ResultsThe patients (n = 16) were divided in a resection group (n = 8) and a transplant group (n = 8). The median age at the time of diagnosis was 13.5 months. The motive for the initial consultation was the discovery of a mass; all patients had high levels of α-fetoprotein and an imaging study. Ten of 16 patients required chemotherapy before the surgical procedure. In the resection group, 5 of 8 patients were classified as Clavien I and 4 of 8 patients of the transplant group were classified as Clavien II. Patient survival at 30 months was 100% in the resection group and 65% in the liver transplantation group.ConclusionsTo our knowledge, this is the first case report of pediatric patients with hepatoblastoma and liver resection or transplant in Colombia and Latin America. Our results are comparable with the series worldwide, showing that resection and transplant increase the survival of the pediatric patients with hepatoblastoma. It is important to advocate for an increase of reporting in the scientific literature in Latin America.
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