Abstract
Objectives: To quantify health related quality of life (HRQOL) of patients with biliary atresia with their native livers and compare them with healthy children and patients with biliary atresia post-liver transplant (LT) and to examine the relationship between HRQOL and medical variables. Study design: A cross-sectional HRQOL study of patients with biliary atresia with their native livers (ages 2-25 years) was conducted and compared with healthy and post-LT biliary atresia samples using Pediatric Quality of Life Inventory 4.0 child self and parent proxy reports, a validated measure of physical/psychosocial functioning. Results: 221 patients with biliary atresia with native livers (54% female, 67% white) were studied. Patient self and parent proxy reports showed significantly poorer HRQOL than healthy children across all domains (P <.001), particularly in emotional and psychosocial functioning. Child self and parent proxy HRQOL scores from patients with biliary atresia with their native livers and post-LT biliary atresia were similar across all domains (P = not significant). Child self and parent proxy reports showed moderate agreement across all scales, except social functioning (poor to fair agreement). On multivariate regression analysis, black race and elevated total bilirubin were associated with lower Total and Psychosocial HRQOL summary scores. Conclusions: HRQOL in patients with biliary atresia with their native livers is significantly poorer than healthy children and similar to children with post-LT biliary atresia. These findings identify significant opportunities to optimize the overall health of patients with biliary atresia.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 1052-1057.e2 |
| Journal | journal of pediatrics |
| Volume | 163 |
| Issue number | 4 |
| DOIs | |
| State | Published - Oct 2013 |
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
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In: journal of pediatrics, Vol. 163, No. 4, 10.2013, p. 1052-1057.e2.
Research output: Contribution to journal › Article › peer-review
TY - JOUR
T1 - Health related quality of life in patients with biliary atresia surviving with their native liver
AU - Sundaram, Shikha S.
AU - Alonso, Estella M.
AU - Haber, Barbara
AU - Magee, John C.
AU - Fredericks, Emily
AU - Kamath, Binita
AU - Kerkar, Nanda
AU - Rosenthal, Philip
AU - Shepherd, Ross
AU - Limbers, Christine
AU - Varni, James W.
AU - Robuck, Patricia
AU - Sokol, Ronald J.
N1 - Funding Information: This multicenter study evaluates HRQOL in a large cohort of children affected by biliary atresia surviving with their native livers and compares them across a spectrum of health, from children who are healthy to those with biliary atresia who have undergone LT, in order to understand the complete impact of this chronic disease. This study captures children with biliary atresia across a broad range of ages and simultaneously reports the perspective of both the child and the parent, providing the most comprehensive assessment of HRQOL in biliary atresia to date. This cohort of patients with biliary atresia was generally healthy, with 62% having a state of health considered by the research team to be optimal for a child with this diagnosis. Their HRQOL, however, was significantly poorer than healthy children. In addition, contrary to our expectations, their HRQOL was surprisingly similar to that of patients with biliary atresia who had previously undergone LT and therefore experienced very different medical problems related to the post-transplant state. Psychosocial functioning in children with biliary atresia surviving with their native livers was significantly impaired compared with a matched healthy population. Psychosocial problems are increasingly identified as “hidden morbidities” across an array of pediatric conditions, now including biliary atresia. 13-15 As such, these findings support the need for psychological counseling and the development of peer support groups for children with biliary atresia. The most significant differences between patients with biliary atresia surviving with their native livers and healthy children occurred in school functioning, a construct that measures missed days of school (which was not assessed in this study) and school-related cognitive functioning. 4,8,16 Infants and toddlers with biliary atresia are known to have subtle delays in gross motor and language development. 17-19 Cognitive and learning disabilities in older children living with biliary atresia, however, have yet to be quantified. Neurodevelopmental testing that is ongoing in patients with biliary atresia participating in the ChiLDREN study may provide further insights into this area in the future. Self reports of social functioning were poorer among young children (ages 5-12 years) than older adolescents and young adults. This finding may imply a “response shift,” whereby over time children accommodate their perceptions of the negative impacts of biliary atresia on their lives. 19-21 Furthermore, modeling that included markers of disease severity did not identify a survivor bias in this study, despite the cross-sectional nature of the study. Future longitudinal analyses of HRQOL in patients with biliary atresia with their native livers may provide additional insight into this finding. Unexpectedly, patients with biliary atresia with their native livers were noted to have nearly identical impairments in HRQOL as patients with biliary atresia who had undergone LT. Seventy-seven biliary atresia subjects with evidence of more advanced liver disease did not, however, complete HRQOL assessments, which may have skewed these results towards better HRQOL. In addition, although the degree of portal hypertension in patients with biliary atresia with their native livers was difficult to quantify based on recorded physical findings, it is expected that many had a degree of portal hypertension, which might contribute to subclinical or minimal hepatic encephalopathy. In adults with cirrhosis with evidence of minimal hepatic encephalopathy on neurocognitive testing, abnormalities improve but are not completely reversed after LT. 22,23 Therefore, this may suggest that cognitive deficits associated with portal hypertension in the pre-transplant period in patients with biliary atresia may still contribute to HRQOL in post-transplant follow-up. Longitudinal assessment of neurocognitive deficits and school performance in children with biliary atresia before and after LT will be necessary to fully understand this evolution in pediatric patients. Self reports by patients with biliary atresia living with their native livers had only moderate agreement with parental HRQOL reports, consistent with previous literature in other chronic diseases. 24,25 The weakest correlation was noted in social functioning, which occurs primarily outside the home in children of school age and beyond and may not be a directly observed behavior. This reiterates that simultaneous evaluation of child and parent perceptions of HRQOL should remain the standard practice, providing complementary but unique perspectives. In this study, black race in patients with biliary atresia was independently associated with poorer HRQOL. This study did not collect data on socioeconomic status, marital status, or education, and, therefore, the potential impact of such confounders cannot be assessed. Higher total bilirubin was also associated with poorer HRQOL in patients with biliary atresia. One might speculate that the addition of the biliary atresia children who did not undergo HRQOL testing and who demonstrated significant cholestasis ( Table I ), could make this association even more robust. Children with more pronounced cholestasis may have more visible signs of disease, including pruritus, nutritional deficiencies, poorer growth, and more frequent medical visits, all of which may contribute to poor HRQOL. A limitation of this study is that 77 biliary atresia subjects and their families chose not to complete the HRQOL assessment. These subjects had evidence of more advanced liver disease. We speculate that these subjects would have had poor overall HRQOL based on the relationship we found between total serum bilirubin and parent proxy-reports of HRQOL. Therefore, our findings in patients with biliary atresia with native livers should be viewed as representing a better outcome than is most likely present in all biliary atresia subjects with their native liver. In conclusion, despite generally good medical health, patients with biliary atresia with their native livers have significantly impaired HRQOL, similar to that in patients with biliary atresia post-LT. Recognition of this allows for the initiation of a dialogue between the care provider, patient, and family designed to promote individualized long-term care in children pre-LT. These findings identify significant deficits in the functioning of patients with biliary atresia that should form the basis for development of comprehensive care plans, with the goal to achieve optimal health. Appendix Members of the Childhood Liver Disease Research and Education Network include: John Hopkins School of Medicine (supported by NIDDK U54DK078377 and DK 62530 and NCRR UL1RR025005) Baltimore, MD: Kathy Schwarz, MD, Robert Anders, MD, PhD, Paul Colombani, MD, Wikrom Karnsakul, MD, Shalom Anita Osire, Kim Pfeifer, RN, BSN, Laura Wachter; Ann & Robert H. Lurie Children’s Hospital of Chicago (supported by NIDDK DK 62436 and NCRR UL1RR025741) Chicago, IL: Peter Whitington, MD, Estella Alonso, MD, Lee Bass, MD, Kelly Dunmars, Denise Jones, Sue Kelly, RN, BSN, Hector Melin-Aldana, MD, Denise Rizza, PharmD, Ricardo Superina, MD, Kiley Tkaczyk, Elizabeth Westfall; Cincinnati Children’s Hospital Medical Center (supported by NIDDK DK 62497 and NCRR UL1RR026314) Cincinnati, OH: Jorge Bezerra, MD, Kevin Bove, MD, Julie Denlinger, James Heubi, MD, Pinky Jha, Denise LaGory, RPH, Alexander Miethke, MD, Joseph Palermo, MD, Stacey Reed, Ken Setchell, PhD, Melissa Stamper, Greg Tiao, MD; Children’s Hospital Colorado (supported by NIDDK DK 62453 and NCRR UL1RR025780) Aurora, CO: Ron Sokol, MD, Megan Dix, Sheryl Faut, Joan Hines, MPH, Michelle Hite, RN, Jessica Jensen, Frederick Karrer, MD, Mark Lovell, MD, Cara Mack, MD, Michael Narkewicz, MD, Timothy Schardt, PharmD, BCPS, Frederick Suchy, MD, Shikha Sundaram, MD, Johan Van Hove, MD; Mount Sinai School of Medicine (supported by NIDDK DK 62445 and NCRR UL1RR029877) New York, NY: Ronen Arnon, MD, Mariel Boyd; Jamie Chu, MD, Ivy Cohen, Kishore Iyer, MD, Margret Magid, MD; The Children’s Hospital of Philadelphia (supported by NIDDK DK 62481 and NCRR UL1RR024134) Philadelphia, PA: Kathleen Loomes, MD, Timothy Crisci, Jessi Erlichman, MPH, Alan Flake, MD, Josh Friedman, MD, PhD, Oyinkansola Kusemiju, MPH, Laura Leonard, David Piccoli, MD, Elizabeth Rand, MD, Pierre Russo, MD, Nancy Spinner, PhD, Eileen Wu; Children’s Hospital of Pittsburgh (supported by NIDDK DK 62466 and NCRR UL1RR024153) Pittsburgh, PA: Benjamin Shneider, MD, Feras Alissa, MD, Beverly Bernard, CRNP, A’Delbert Bowen, MD, Kathy Bukauskas, RN, CCRC, Paul Hoffmann, Ronald Jaffe, MD, Douglas Lindblad, MD, George Mazariegos, MD, Robert Ortiz-Iguayo, MD, David Perlmutter, MD, Stefan Scholz, MD, Rakesh Sindhi, MD, Robert Squires, Jr., MD, Veena Venkat, MD, Jerry Vockley, MD, PhD, Dale Zecca; UCSF Children’s Hospital (supported by NIDDK DK 62500 and NCRR UL1RR024131) San Francisco, CA: Philip Rosenthal, MD, Laura Bull, PhD, Scott Fields, Shannon Fleck, Melvin Heyman, MD, Shinjiro Hirose, MD, Vincent Hoang, Grace Kim, MD, Camille Langlois, John Pech, Claudi Tejeda, John Roberts, MD, Larry Wang, MD, PhD; Washington University School of Medicine (Site 9 - supported by NIDDK DK 62452 and NCRR UL1RR024992) St. Louis, MO: Yumi Turmelle, MD, Pat Dillon, MD, Sandra Guelker, BA, Kathleen Harris, Robert Heuckeroth, MD, Jeffrey Lowell, MD, FACS, Stacy Postma, Jonathan (Brad) Rider, RPH, David Rudnick, MD, PhD, Janis Stoll, Alexander Weymann, MD, Frances White, MD; Texas Children’s Hospital (supported by NIDDK DK 62470) Houston, TX: Paula Hertel, MD, Zoe Apted, BA, Mary Brandt, MD, Jameisha Brown, Beth Carter, MD, Milton Finegold, MD, Richard Gibbs, PhD, Sanjiv Harpavat, MD, Jennifer Lynds, RPh, PharmD, Tara McCartney, RPh; St. Louis University School of Medicine, St. Louis, MO: Jeffrey Teckman, MD, Vikki Kociela, BSN, CCRC; Riley Hospital for Children, Indianapolis, IN: Jean Molleston, MD, Molly Bozic, MD, Beth Byam, RN, Oscar (Bill) Cummings, MD, Vicki Haviland, Ann Klipsch, RN, Cindy Sawyers, BSRT, Girish Subbarao, MD, Jada Wegere, Karen West; Seattle Children’s Hospital, Seattle, WA: Karen Murray, MD, Kara Cooper, Laura Finn, MD, Patrick Healey, MD, Simon Horslen, MD, Susan Jacob, Alexander Miethke, MD, Joseph Palermo, MD, Stacey Reed, Ken Setchell, PhD, Melissa Stamper, MD; The Hospital for Sick Children, Toronto, Ontario, CA: Vicky Ng, MD, Allison Chiu, Catherine Chung, MD, Catherine Day, Maria DeAngelis, Annie Fecteau, MD, Kelsey Hunt, Binita Kamath, MD, Tanya Kovaleva, Jacob Langer, MD, Constance O’Connor, Claudia Quammie, Lilatool Shakur, Krista VanRoestel; Children’s Hospital Los Angeles, Los Angeles, CA: Kasper Wang, MD, JoAnna Cavallero, Henri Ford, MD, MHA, Catherine Goodhue, CPNP, Nanda Kerker, MD, Sonia Michail, MD, Vincent Hoang, Grace Kim, MD, Camille Langlois, John Pech, John Roberts, MD, Larry Wang, MD, PhD; Children’s Healthcare of Atlanta (Site P [18]) Atlanta, GA: Saul Karpen MD, PhD, Carlos Abramowsky, MD, Liezl de la Cruz-Tracy, Rose Francois-Marcello, Nitika Gupta, MD, Jim Rhodes, PharmD, Richard Ricketts, MD, Rene Romero, MD, Katrina Scott, Sundari Sekar, MBBS, DGO, Bahig Shehata, MD, Taieshia Turner-Green, Miriam Vos, MD, MSPH; King’s College Hospital, London, UK: Richard Thompson National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD: Patricia Robuck, PhD, Edward Doo, MD, Jay Hoofnagle, MD, Averell Sherker, MD, FRCP, Rebecca Torrance, RN, MSN, Rebekah Van Raaphorst, MPH, LT; University of Michigan Data Coordinating Center (supported by NIDD DK 62456) Ann Arbor, MI: John Magee, MD, Karen Jones, Beverley Marchant, Robert, ScD Parker, Trivellore Raghunathan, PhD, Cathie Spino, DSc, Wen Ye. Table II A comparison of child-self and parent-proxy reports of PedsQL 4.0 Generic Core Scale scores in biliary atresia patients with their native livers vs healthy children and children with biliary atresia after LT Scale Biliary atresia native liver Healthy sample Differences Effect size Biliary atresia post-LT Differences Effect size Mean SD Mean SD Mean SD Child self-report (n = 173) (n = 954) (n = 151) Total score 76.96 13.70 84.70 12.16 7.74 ∗ 0.62 75.22 14.56 1.74 0.12 Physical score 83.41 14.99 88.86 12.31 5.45 ∗ 0.43 81.15 16.38 2.26 0.14 Psychosocial score 73.51 15.53 82.49 14.03 8.98 ∗ 0.63 71.95 15.97 1.56 0.10 Emotional functioning 70.92 20.96 80.56 18.08 9.64 ∗ 0.52 71.28 19.07 0.36 0.02 Social functioning 80.49 16.59 85.51 16.82 5.02 ∗ 0.30 78.96 20.92 1.53 0.08 School functioning 69.13 20.26 81.25 16.49 12.12 ∗ 0.71 65.77 17.47 3.36 0.18 Parent proxy-report (n = 197) (n = 1266) (n = 437) Total score 77.69 16.40 84.58 13.30 6.89 ∗ 0.50 78.05 16.83 0.36 0.02 Physical score 82.04 21.50 87.28 16.35 5.24 ∗ 0.31 82.22 16.92 0.18 0.01 Psychosocial score 75.28 15.91 83.10 13.78 7.82 ∗ 0.56 75.66 17.25 0.38 0.02 Emotional functioning 73.74 17.08 81.86 16.01 8.12 ∗ 0.50 73.70 19.42 0.04 0.00 Social functioning 80.94 18.37 86.49 16.75 5.55 ∗ 0.33 80.97 20.19 0.03 0.00 School functioning 68.35 20.39 80.64 17.69 12.29 ∗ 0.68 69.73 21.51 1.38 0.07 ∗ P < .001 based on independent samples t -tests. Effect sizes are designated as small (0.20), medium (0.50), and large (0.80).
PY - 2013/10
Y1 - 2013/10
N2 - Objectives: To quantify health related quality of life (HRQOL) of patients with biliary atresia with their native livers and compare them with healthy children and patients with biliary atresia post-liver transplant (LT) and to examine the relationship between HRQOL and medical variables. Study design: A cross-sectional HRQOL study of patients with biliary atresia with their native livers (ages 2-25 years) was conducted and compared with healthy and post-LT biliary atresia samples using Pediatric Quality of Life Inventory 4.0 child self and parent proxy reports, a validated measure of physical/psychosocial functioning. Results: 221 patients with biliary atresia with native livers (54% female, 67% white) were studied. Patient self and parent proxy reports showed significantly poorer HRQOL than healthy children across all domains (P <.001), particularly in emotional and psychosocial functioning. Child self and parent proxy HRQOL scores from patients with biliary atresia with their native livers and post-LT biliary atresia were similar across all domains (P = not significant). Child self and parent proxy reports showed moderate agreement across all scales, except social functioning (poor to fair agreement). On multivariate regression analysis, black race and elevated total bilirubin were associated with lower Total and Psychosocial HRQOL summary scores. Conclusions: HRQOL in patients with biliary atresia with their native livers is significantly poorer than healthy children and similar to children with post-LT biliary atresia. These findings identify significant opportunities to optimize the overall health of patients with biliary atresia.
AB - Objectives: To quantify health related quality of life (HRQOL) of patients with biliary atresia with their native livers and compare them with healthy children and patients with biliary atresia post-liver transplant (LT) and to examine the relationship between HRQOL and medical variables. Study design: A cross-sectional HRQOL study of patients with biliary atresia with their native livers (ages 2-25 years) was conducted and compared with healthy and post-LT biliary atresia samples using Pediatric Quality of Life Inventory 4.0 child self and parent proxy reports, a validated measure of physical/psychosocial functioning. Results: 221 patients with biliary atresia with native livers (54% female, 67% white) were studied. Patient self and parent proxy reports showed significantly poorer HRQOL than healthy children across all domains (P <.001), particularly in emotional and psychosocial functioning. Child self and parent proxy HRQOL scores from patients with biliary atresia with their native livers and post-LT biliary atresia were similar across all domains (P = not significant). Child self and parent proxy reports showed moderate agreement across all scales, except social functioning (poor to fair agreement). On multivariate regression analysis, black race and elevated total bilirubin were associated with lower Total and Psychosocial HRQOL summary scores. Conclusions: HRQOL in patients with biliary atresia with their native livers is significantly poorer than healthy children and similar to children with post-LT biliary atresia. These findings identify significant opportunities to optimize the overall health of patients with biliary atresia.
UR - http://www.scopus.com/inward/record.url?scp=84884669530&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84884669530&partnerID=8YFLogxK
U2 - 10.1016/j.jpeds.2013.04.037
DO - 10.1016/j.jpeds.2013.04.037
M3 - Article
C2 - 23746866
AN - SCOPUS:84884669530
SN - 0022-3476
VL - 163
SP - 1052-1057.e2
JO - journal of pediatrics
JF - journal of pediatrics
IS - 4
ER -