Observational cohort study of long-term outcomes of liver transplantation in haemophilia.

INTRODUCTION: Gene therapy is now a reality for individuals with haemophilia, yet little is known regarding the quality-of-life impact of factor correction. As few data exist, and recognizing the analogy to liver transplantation (OLTX), we identified OLTX+ and OLTX- men in the ATHNdataset to compare post-OLTX factor VIII and IX on quality of life (QoL) by Haem-A-QoL and PROMIS-29. METHODS: OLTX- were matched to OLTX+ by age, race, and haemophilia type and severity. Deidentified demographic data, including post-transplant factor levels, genotype and target joint disease were analysed by descriptive statistics. Haem-A-Qol and PROMIS-29 were compared in OLTX+ and OLTX- by student's t-test and univariate regression models. RESULTS: Of 86 people with haemophilia A (HA) or haemophilia B (HB) cared for at 10 haemophilia treatment centers (HTCs), 21 (24.4%) OLTX+ and 65 (75.6%) OLTX- were identified. OLTX+ and OLTX- had a similar frequency of target joint disease (p = .806), HA genotypes, null versus non-null (p = .696), and HIV infection (p = .316). At a median 9.2 years post-OLTX, median FVIII, .63 IU/mL [IQR 0.52-0.97] and FIX, .91 IU/mL [IQR .63-1.32], Haem-A-QoL, PROMIS-29, and HOT scores were comparable. Severe HA/HB had lower post-OLTX 'dealing with haemophilia' scores (p = .022) and higher 'sports and leisure' (p = .010) and 'view of yourself' scores (p = .024) than OLTX+ non-severe participants. Non-caucasian OLTX+ had significantly lower scores in sports and leisure (p = .042), future expectations (p = .021) and total score (p = .010). CONCLUSION: Nine years after OLTX, QoL is comparable to OLTX-, but significantly better in OLTX+ with severe than non-severe disease and in caucasians than non-caucasians.

Comparison of Alvarado score evaluation and clinical judgment in acute appendicitis.

BACKGROUND: Acute appendicitis is the most common surgical emergency in children, but its diagnosis is sometimes difficult. The aim of this study is to evaluate retrospectively the Alvarado score in relation to the surgical management based on clinical judgment. METHODS: Medical files of 232 children who underwent appendectomy at Makassed General Hospital from January 1997 till December 2006 were reviewed. Demographic characteristics, symptoms and signs, laboratory results and imaging findings for all children were recorded. RESULTS: The positive predictive value of our clinical judgment was 86.4% and the negative appendectomy rate was 13.6% based on the pathology results. The reliability of Alvarado score in our population found a PPV of 80.7% and a negative appendectomy rate of 11.3%. A multivariate analysis revealed that anorexia, neutrophils left shift and rebound tenderness are significantly correlated with a correct diagnosis of appendicitis (p = 0.012, 0.023 and 0.046 respectively). CONCLUSION: Although, Alvarado score provides measurably useful diagnostic information in evaluating children with suspected appendicitis, we found that good clinical judgment remain the main stay of correct diagnosis of appendicitis.

The effectiveness of pudendal nerve block versus caudal block anesthesia for hypospadias in children.

BACKGROUND: Caudal block (CB) has some disadvantages, one of which is its short duration of action after a single injection. For hypospadias repair, pudendal nerve block (PNB) might be a suitable alternative since it has been successfully used for analgesia for circumcision. We evaluated PNB compared with CB as measured by total analgesic consumption 24 hours postoperatively. METHODS: In this prospective, double-blinded study, patients were randomized into 2 groups, either receiving CB or nerve stimulator-guided PNB. In the PNB group, patients were injected with 0.3 mL/kg 0.25% bupivacaine and 1 µg/kg clonidine. In the CB group, patients were injected with 1 mL/kg 0.25% bupivacaine and 1 µg/kg clonidine. Analgesic consumption was assessed during the first 24 hours postoperatively. The "objective pain scale" developed by Hannalah and Broadman was used to assess postoperative pain. RESULTS: Eighty patients participated in the study, 40 in each group. The mean age in the PNB group was 3.1 (1.1) years and in the CB group was 3.2 (1.1) years. The mean weights in the PNB and CB groups were 15.3 (2.8) kg and 15.3 (2.2) kg, respectively. The percentage of patients who received analgesics during the first 24 hours were significantly higher in the CB (70%) compared with the PNB group (20%, P < 0.0001). The average amount of analgesics consumed per patient within 24 hours postoperatively was higher in the CB group (paracetamol P < 0.0001, Tramal P =0.003). CONCLUSION: Patients who received PNB had reduced analgesic consumption and pain within the first 24 hours postoperatively compared with CB.