Introduction: Microvillus inclusion disease (MVID) is a rare autosomal recessive congenital defect of the enterocyte that causes intractable diarrhea leading to life-threatening dehydration and malnutrition in early infancy. Current management and treatment consist of total parenteral nutrition as a bridge to eventual intestinal transplantation. There has been some debate on the best time for transplantation. This study aimed to compare nutritional and patient survival outcomes by transplant status and timing of transplantation.
Methods: This retrospective chart review included MVID patients aged 0-19 years at the time of initial evaluation at our institution from 1/1994 to 11/2019. Data were collected for patient demographics, clinical characteristics, transplant-related information, and nutritional status. Groups of children by transplant status and by their age at transplant (< 2 years, 2 to < 5 years, and ³ 5 years) were compared using Fisher's exact test for categorical variables and using Kruskal-Wallis test for continuous variables. The Kaplan-Meier method was performed to estimate the patient survival, using the log-rank chi-square test to compare the curves between the groups.
Results: Twenty-five children (16 males, 19 transplanted, 17 liver-small bowel transplants) were included. Among transplant children, 11 were transplanted prior to 2 years, 5 were transplanted from age 2 to 5 years, and 3 were transplanted after age 5 years. The median (interquartile range) length of follow-up was 3.8 (8.4) years in the transplant group versus 0.8 (1.3) years in the non-transplant group (p =0.006). There was no significant difference in the median length of follow-up between the three transplant groups. All transplant patients achieved enteral autonomy at the time of transplant discharge; the majority of them remained on full enteral and/or oral feeds at the last follow-up. Table 1 presents nutritional parameters by transplant status and by age at transplant. Overall, 14 (73.7%) transplant and 3 (50%) non-transplant patients died (p=0.34), with infection being the leading cause of death. Three transplant children required re-transplantation. Figure 1 compares the overall patient survival between the transplant groups (log-rank test, p=0.76).
Conclusions: While the overall patient survival was not statistically significant different between the transplant age groups, nutritional outcomes appeared to be negatively impacted with later transplant. Early transplant may offer benefits of early feeding therapy to avoid oral aversion and provide more opportunity for catch-up growth. A future, larger scale, multicenter study would be beneficial in defining the optimal timing for transplant to improve nutritional and patient survival outcomes.