Nutritional status and prognosis in children with immunodeficiencies undergoing hematopoietic stem cell...

Primary immunodeficiencies (PID) are diseases resulting from genetic dysfunctions in the immune system, which can result in recurrent infections, autoimmunity and even malignancy. It is estimated that approximately one-third of the PID described have gastrointestinal components or symptoms involved and may present an increased risk of weight loss and failure to thrive. It is also known that, in patients with other diagnoses, malnutrition may be associated with worse outcomes after hematopoietic stem cell transplantation (HSCT).

to characterize the nutritional status of pediatric patients with PID at the time of admission for HSCT and to establish the relationship between baseline nutritional status measures and post-HSCT clinical outcomes.

a retrospective analytical observational study, based on data from pediatric patients, of both sexes and all ethnicities, with PID, submitted to HSCT in the period from 2004 to 2019. The risk factors analyzed were the Z-scores of weights for age (W/A), height for age (H/A), BMI for age (BMI/A) and Sum score, obtained by through the sum of the W/A and H/A scores. The primary outcomes were overall survival at 6 months, occurrence of acute Graft Versus Host Disease (aGVHD) at 6 months, and occurrence of chronic Graft Versus Host Disease (cGVHD) at 1 year. Secondary outcomes were occurrence and degree of mucositis, length of stay, and total number of infectious episodes. As statistical analysis, the ANOVA model, the Tukey test, ROC curves and Kaplan Meier and Log-Rank analysis were used. Multivariate survival and logistic regression models were also performed.

The study showed important indicators of malnutrition in patients with PID, especially those diagnosed with Severe Combined Immunodeficiency Syndrome (SCID) and Hemophagocytic Syndromes (HS). Among those with SCID, 60% had low or very low weight for their age, 52% had low or very short stature for their age, and 44% were classified as being thin or very thin. Among patients with HS, 75% had short or very short stature for their age. Multivariate analysis only demonstrated association between W/A score with extensive cGVHD, controlling for diagnosis, compatibility, conditioning and immunoprophylaxis. Lower W/A values were associated with higher occurrences of these events. Although W/A was only associated with cGVHD and H/A had no association with chronic or acute GVHD, when Sum scores were used, the lower values the higher rates of severe aGVHD and total cGVHD according to multivariate controlled models for diagnosis, compatibility, conditioning and immunoprophylaxis.

Our study characterized the nutritional status of children with PID undergoing HSCT and found alarming rates of underweight and short stature in patients with SCID and HS. We also demonstrated a relationship between anthropometric parameters and outcomes such as mortality, the occurrence of GVHD and severe mucositis after HSCT. In this sense, W/A and Sum score measures would be good prognostic methods for these outcomes. Henceforth, prospective studies are needed to confirm these findings and establish new nutritional assessment criteria for this population.