Outcomes of Cardiac Transplantation in Highly Sensitized Pediatric Patients

Despite aggressive immunosuppressive therapy, pediatric orthotopic heart transplant (OHT) candidates with elevated pre-transplant panel reactive antibody (PRA) carry an increased risk of rejection and early graft failure following transplantation. This study has aimed to more specifically evaluate the outcomes of transplant candidates stratified by PRA values. Records of pediatric patients listed for OHT between April 2004 and July 2008 were reviewed (n = 101). Survival analysis was performed comparing patients with PRA < 25 to those with PRA > 25, as well as patients with PRA < 80 and PRA > 80. Patients with PRA > 25 had decreased survival compared with those with PRA < 25 after listing (P = 0.004). There was an even greater difference in survival between patients with PRA > 80 and those with PRA < 80 (P = 0.002). Similar analyses for the patients who underwent successful transplantation showed no significant difference in post-transplant survival between patients with a pre-transplant PRA > 25 and those with PRA < 25 (P = 0.23). A difference approaching significance was noted for patients with PRA > 80 compared with PRA < 80 (P = 0.066). Patients with significantly elevated pre-transplant PRAs at the time of listing have a significantly worse outcome compared to those with moderately increased PRA values or non-sensitized patients. Further study is necessary to guide physician and family treatment decisions at the time of listing

Measuring Health Utilities in Children and Adolescents: A Systematic Review of the Literature.

BACKGROUND: The objective of this review was to evaluate the use of all direct and indirect methods used to estimate health utilities in both children and adolescents. Utilities measured pre- and post-intervention are combined with the time over which health states are experienced to calculate quality-adjusted life years (QALYs). Cost-utility analyses (CUAs) estimate the cost-effectiveness of health technologies based on their costs and benefits using QALYs as a measure of benefit. The accurate measurement of QALYs is dependent on using appropriate methods to elicit health utilities. OBJECTIVE: We sought studies that measured health utilities directly from patients or their proxies. We did not exclude those studies that also included adults in the analysis, but excluded those studies focused only on adults. METHODS AND FINDINGS: We evaluated 90 studies from a total of 1,780 selected from the databases. 47 (52%) studies were CUAs incorporated into randomised clinical trials; 23 (26%) were health-state utility assessments; 8 (9%) validated methods and 12 (13%) compared existing or new methods. 22 unique direct or indirect calculation methods were used a total of 137 times. Direct calculation through standard gamble, time trade-off and visual analogue scale was used 32 times. The EuroQol EQ-5D was the most frequently-used single method, selected for 41 studies. 15 of the methods used were generic methods and the remaining 7 were disease-specific. 48 of the 90 studies (53%) used some form of proxy, with 26 (29%) using proxies exclusively to estimate health utilities. CONCLUSIONS: Several child- and adolescent-specific methods are still being developed and validated, leaving many studies using methods that have not been designed or validated for use in children or adolescents. Several studies failed to justify using proxy respondents rather than administering the methods directly to the patients. Only two studies examined missing responses to the methods administered with respect to the patients' ages

Emerging clinical picture of lymphangioleiomyomatosis

Objective: To provide a comprehensive update of the clinical picture of lymphangioleiomyomatosis (LAM) using two large patient registries. Methods: A cross sectional questionnaire survey which included questions on 14 LAM symptoms, pneumothorax, tuberous sclerosis complex (TSC), date of diagnosis, and pulmonary function tests (PFTs). Results: The response rate was 70.5% (n = 328). The mean age at the time of the survey was 46.7 years. The mean age at diagnosis was 42.7 years for women diagnosed 1 year before the survey and 35.8 years for women diagnosed 10 years previously. The main symptoms were dyspnoea (74%), fatigue (72%), cough (47%) and chest pain (44%); younger patients (<40 years) were less likely to report dyspnoea (p = 0.02). Patients with TSC (n = 51) were less likely to report dyspnoea (p = 0.05) and 76.5% reported angiomyolipoma (p<0.0001) compared with patients with sporadic LAM. Patients with pneumothorax (63.0%) were less likely to report dyspnoea or fatigue (p⩽0.05) than patients without pneumothorax. PFT results showed that low forced expiratory volume in 1 second and carbon monoxide transfer factor were highly associated with dyspnoea (p<0.0001), but not with fatigue or history of pneumothorax. Conclusion: Previously considered a condition of women of childbearing age, more older women (50% without pneumothorax) are now being diagnosed with LAM. LAM should be considered in women over 40 with unexplained dyspnoea. LAM patients with pneumothorax have less fatigue and less dyspnoea than those without pneumothorax. Fatigue has been overlooked as a symptom of LAM and appears across the spectrum of pulmonary function

Canadian Respirologists’ Experience with Lymphangioleiomyomatosis

BACKGROUND: Lymphangioleiomyomatosis (LAM) is a rare pulmonary disease occurring primarily in women. A literature review of LAM in Canada found sporadic mention of LAM in case reports or within lung transplant studies. The LAM Foundation, a patient support and research funding organization, lists 23 Canadian patients in its database. The present study was designed to assess the scope of LAM across Canada and to identify potential patients for further evaluation. OBJECTIVES: To ascertain Canadian respirologists’ experience with patients with LAM (current and historical), lung transplantation (single or bilateral) and deaths due to LAM, and awareness of the LAM Foundation.METHODS: Four hundred twelve brief surveys were sent anonymously to members of the Canadian Lung Association (inserted in their newsletters) to ascertain the experience of Canadian respirologists with LAM.RESULTS: One hundred twelve surveys were returned (27%). Fifty-one respondents had 'ever' been involved in the care of at least one patient with LAM; eight had cared for three or more patients. At the time of the study, 26 respondents were following a total of 46 patients with LAM; 22 of the 51 respirologists (43%) who had ever cared for a patient with LAM reported the death of a patient. Thirty-three patients had been put on wait lists for transplantation; six died while on the wait list. Nineteen patients underwent lung transplantation - six single-lung recipients and 13 bilateral lung recipients. Of the 51 respirologists who had ever cared for a patient with LAM, only 30 (61%) were aware of the LAM Foundation’s services. Of the 112 respondents, only 47 (43%) were aware of the LAM Foundation.CONCLUSIONS: This study identified a moderate level of awareness of a significant existing patient support and research service (the LAM Foundation). There were many patients with LAM who were unknown to the LAM Foundation and could benefit from its resources. Results suggest that there may be more patients with LAM in Canada than are reported in the existing literature.Peer Reviewe