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The purpose of this study was to determine perioperative risk factors for need of liver transplantation following hepatoportoenterostomy. A retrospective review of patients undergoing hepatoportoenterostomy for biliary atresia at our institution from 1990 to 2016 was completed. A total of 81 patients were identified with a median age of 51 days (IQR: 33-68) at hepatoportoenterostomy and a median follow-up time of 5.7 years (IQR: 1-11.6). Ten-year overall survival was 93% (95% CI: 84-97). Thirty-six patients (44%) ultimately required transplantation at a median time from hepatoportoenterostomy of 8.9 months (IQR: 5.2-19). The 10-year transplant-free survival was 36% (95%CI: 24-49). Steroid use (N = 42) was not associated with improved 10-yr transplant-free survival (33% vs. 38%, p = 0.690). Age at hepatoportoenterostomy was not significantly associated with the need for transplantation. Multivariable logistic regression analysis demonstrated that total bilirubin > 2mg/dL (OR: 97, p < 0.001) and albumin < 3.5g/dL (OR: 24, p = 0.027) at 3 months after surgery were independent predictors of the need for transplantation, while adjusting for age, sex, prematurity, and steroid use. Overall survival for children with biliary atresia is excellent, although most patients will ultimately require liver transplantation. Total bilirubin and albumin level at 3 months following hepatoportoenterostomy are predictive of the need for transplantation. Steroid use is not associated with improved outcomes. [ABSTRACT FROM AUTHOR]

Background Dedicator of cytokinesis 8 (DOCK8) deficiency can be cured by allogeneic hematopoietic stem cell transplantation (HSCT). Reports of outcomes are still limited. Objective We sought to analyze the results of HSCT in patients with DOCK8 deficiency and report whether approaches resulting in mixed chimerism result in clinically relevant immune reconstitution. Methods We performed a retrospective chart review of 11 patients with DOCK8 deficiency and measured DOCK8 expression and cytokine production. Results Of 11 patients, 7 received HSCT from related and 4 from unrelated donors; 9 patients received busulfan-based conditioning regimens. Survival was excellent (10 [91%] of 11 patients alive), including a patient who had undergone liver transplantation. Patients showed significant improvements in the frequency and severity of infections. Although eczema resolved in all, food allergies and high IgE levels persisted in some patients. Lymphopenia, eosinophilia, low numbers of naive CD8 + T cells and switched memory B cells, and T H 1/T H 2 cytokine imbalance improved in most patients. Although the 8 matched related or unrelated donor recipients had full donor chimerism, all 3 recipients of mismatched unrelated donor HSCT had high levels of donor T-cell chimerism and low B-cell and myeloid cell chimerism (0% to 46%). Almost all switched memory B cells were of donor origin. All patients, including those with mixed chimerism, mounted robust antibody responses to vaccination. Conclusion Allogeneic HSCT ameliorated the infectious and atopic symptoms of patients with DOCK8 deficiency. In patients with mixed chimerism, selective advantage for donor-derived T cells and switched memory B cells promoted restoration of cellular and humoral immunity and protection against opportunistic infection. [ABSTRACT FROM AUTHOR]