The first randomized controlled trial in pediatric nephrology: the history of the International Study of Kidney Disease in Children (ISKDC).

The International Study of Kidney Disease in Children (ISKDC), begun in 1966, conducted the first international collaborative randomized blinded controlled trial in pediatric nephrology and one of the first in either pediatrics or nephrology. For this trial, the ISKDC developed the criteria, such as those for response and relapse, used today to describe the clinical course of the nephrotic syndrome, and the trial generated the nephropathologic terminology and criteria which largely remain the current standards. Over an approximately 20-year span, the ISKDC followed the natural history and evaluated the therapeutic effectiveness of therapies in over 500 children with the nephrotic syndrome from three continents. It published 14 peer-reviewed studies and several reports and commentaries, many of which helped create current standards of practice for therapy of childhood nephrotic syndrome and consequently remain highly cited today. The ISKDC continues to be an important model for subsequent collaborative studies and was the impetus for the development of regional and national pediatric nephrology societies leading to the recognition and growth of pediatric nephrology as a separate subspecialty. A higher resolution version of the Graphical abstract is available as Supplementary information.

Utility of sedation for young children undergoing dimercaptosuccinic acid renal scans.

BACKGROUND: No studies have examined whether use of sedation during a Tc-99 m dimercaptosuccinic acid (DMSA) renal scan reduces patient discomfort. OBJECTIVE: To compare discomfort level during a DMSA scan to the discomfort level during other frequently performed uroradiologic tests, and to determine whether use of sedation during a DMSA scan modifies the level of discomfort. MATERIALS AND METHODS: We examined the discomfort level in 798 children enrolled in the Randomized Intervention for children with Vesicoureteral Reflux (RIVUR) and Careful Urinary Tract Infection Evaluation (CUTIE) studies by asking parents to rate their child's discomfort level with each procedure on a scale from 0 to 10. We compared discomfort during the DMSA scan and the DMSA image quality between centers in which sedation was used >90% of the time (sedation centers), centers in which sedation was used <10% of the time (non-sedation centers), and centers in which sedation was used on a case-by-case basis (selective centers). RESULTS: Mean discomfort level was highest for voiding cystourethrogram (6.4), followed by DMSA (4.0), followed by ultrasound (2.4; P<0.0001). Mean discomfort level during the DMSA scan was significantly higher at non-sedation centers than at selective centers (P<0.001). No difference was apparent in discomfort level during the DMSA scan between sedation centers and selective centers (P=0.12), or between the sedation centers and non-sedation centers (P=0.80). There were no differences in the proportion with uninterpretable DMSA scans according to sedation use. CONCLUSION: Selective use of sedation in children 12-36 months of age can reduce the discomfort level experienced during a DMSA scan.

Sequential renal and bone marrow transplants in a child with Fanconi anemia.

FA is an autosomal recessive disorder characterized by small stature and renal abnormalities. FA can lead to progressive bone marrow failure, myelodysplastic syndrome, or acute leukemia. Using a multidisciplinary team approach, we managed a 3-yr-old boy with FA who simultaneously developed renal and hematopoietic failure. Because renal function was insufficient to support the conditioning regimen for HCT, we performed a deceased donor renal transplant in December 2012 prior to HCT with the known risk of graft-versus-graft rejection of the donor kidney. Seven months later he underwent allogeneic HCT. He obtained myeloid engraftment on day +11 and peripheral blood chimerism demonstrated all donor by day +21. He developed asymptomatic CMV reactivation and despite antirejection medications, mild skin graft-versus-host disease. He has maintained excellent renal function and remains transfusion independent with full hematopoietic recovery. He has not experienced any renal rejection episodes nor developed donor-specific antibodies toward his renal donor. Peripheral blood chimerism remains completely HCT donor. He is clinically well, now greater than two and a half yr after renal transplant and two yr after HCT. The continuing close collaboration between the Pediatric Nephrology and Bone Marrow Transplant teams is a major factor in this successful outcome.

The US pediatric nephrology workforce: a report commissioned by the American Academy of Pediatrics.

The US pediatric nephrology workforce is poorly characterized. This report describes clinical and nonclinical activities, motivations and disincentives to a career in pediatric nephrology, future workforce needs, trainee recruitment, and possible explanations for personnel shortages. An e-mail survey was sent in 2013 to all identified US-trained or -practicing pediatric nephrologists. Of 504 respondents, 51% are men, 66% are US graduates, and 73% work in an academic setting. About 20% of trained pediatric nephrologists no longer practice pediatric nephrology. Among the 384 respondents practicing pediatric nephrology full or part-time in the United States, the mean work week was 56.1±14.3 hours, with time divided between patient care (59%), administration (13%), teaching (10%), clinical research (9%), basic research (6%), and other medical activities (3%). Most (>85%) care for dialysis and transplantation patients. The median number of weeks annually on call is 16, and 29% work with one or no partner. One-third of US pediatric nephrologists (n=126) plan to reduce or stop clinical nephrology practice in the next 5 years, and 53% plan to fully or partially retire. Almost half the division chiefs (47%) report inadequate physician staffing. Ongoing efforts to monitor and address pediatric nephrology workforce issues are needed.

Predictors of relapse and end stage kidney disease in proliferative lupus nephritis: focus on children, adolescents, and young adults.

BACKGROUND AND OBJECTIVES: The prevalence and significance of remission and relapse in children, adolescents, and young adults with lupus nephritis in the United States are poorly understood. Patterns and predictors of disease progression in a southeastern U.S. pediatric cohort with severe lupus nephritis are presented. DESIGN, SETTINGS, PARTICIPANTS, & MEASUREMENTS: Individuals age 21 or less with kidney biopsy-proven lupus nephritis followed in the Glomerular Disease Collaborative Network were included. Cox regression models were used to evaluate predictors of relapse and end stage kidney disease (ESKD). RESULTS: Seventy-three subjects with a mean age of 15.6 +/- 3.4 yr were included. Five-year kidney survival was 77%. Complete and partial remission rates within 1 yr of induction therapy were 25 and 64%, respectively. Relapse and ESKD rates were similar between complete and partial responders. Relapse occurred in 35% of responders (complete or partial) in 45 +/- 32 mo. Disease relapse was a predictor of ESKD (HR = 10.12, P < 0.0001). Treatment resistance was documented in African Americans more often than non-African Americans (eight versus 0; P = 0.03). ESKD HR associated with treatment resistance was 6.25, P < 0.002. CONCLUSIONS: Remission whether complete or partial is associated with improved kidney survival in children with lupus nephritis. Nephritis relapse is a strong predictor of progression to ESKD. Treatment resistance portends a high risk of ESKD and disproportionately affects African American children with lupus nephritis.

Parents as information intermediaries between primary care and specialty physicians.

OBJECTIVE: In this study we assessed the views of parents of children referred to specialty care and the views of the children's primary care and specialty physicians about parents' roles as information intermediaries. METHODS: We enrolled 179 patients who were newly referred from primary care pediatricians in 22 practices to 15 pediatric subspecialists in 5 specialties in a study of primary care pediatrician-specialist communication. Parents, primary care pediatricians, and specialists completed questionnaires by mail or telephone at the first visit and 6 months later. Questions included perceived responsibilities of parents as information conduits between primary care pediatricians and specialists. Opinions of parents, primary care pediatricians, and specialists about parents' roles were compared for the sample as a whole, as well as for individual cases. Agreement between parents and providers was assessed. Demographic and clinical determinants of parents reporting themselves as "comfortable with" or "acting" as primary intermediaries were assessed using logistic regression. RESULTS: More parents (44%) than primary care physicians (30%) felt comfortable with parents acting as primary communicators between their children's physicians; 31% of parents who reported that they were the primary communicators felt uncomfortable in that role, and there was no agreement between parents and physicians about the role of parents in individual cases. Although no demographic characteristics of children or parents were associated with parent comfort as the primary communicator, parents of children who saw the same specialist more than once during the 6-month period felt more comfortable in this role. The presence of a chronic condition was not associated with parent comfort. CONCLUSIONS: Although parents report more comfort with their own ability as information intermediaries than do their children's physicians, the role in which they feel comfortable is highly variable. Physicians should discuss with parents the roles they feel comfortable in assuming when specialty referrals are initiated.

Determinants and impact of generalist-specialist communication about pediatric outpatient referrals.

OBJECTIVE: Effective communication between primary care and specialty physicians is essential for comanagement when children are referred to specialty care. We sought to determine rates of physician-reported communication between primary care physicians and specialists, the clinical impact of communication or its absence, and patient- and practice system-level determinants of communication for a cohort of children referred to specialty care. METHODS: We enrolled 179 patients newly referred from general pediatricians in 30 community practices to 15 pediatric medical specialists in 5 specialties. Primary care physicians and specialists completed questionnaires at the first specialty visit and 6 months later. Questions covered communication received by primary care physicians and specialists, its impact on care provision, system characteristics of practices, and roles of physicians in treatment. We used multivariate logistic regression to determine associations between practice system and patient characteristics and the dependent variable of reported primary care physician-specialist communication. RESULTS: Specialists reported communication from referring primary care physicians for only 50% of initial referrals, whereas primary care physicians reported communication from specialists after 84% of initial consultations. Communication was strongly associated with physicians' reported ability to provide optimal care. System characteristics associated with reported primary care physician-specialist communication were computer access to chart notes and lack of delays in receipt of information. Associated patient characteristics included non-Medicaid insurance, no additional specialists seen, and specialty to which referred. Physicians favored comanagement of referred patients in more than two thirds of the cases. CONCLUSIONS: Although a prerequisite for optimal care, communication from primary care physicians to specialists is frequently absent. Interventions should promote widely accessible clinical information systems and target children with complex needs and public insurance.

Generalist-subspecialist communication for children with chronic conditions: a regional physician survey.

OBJECTIVE: To identify target areas for interventions to improve communication between pediatric generalists (PCPs) and pediatric subspecialists (SPs) in the outpatient care of children with chronic conditions. METHODS: We constructed a 4-page mailed questionnaire probing communication practices, opinions about the role of communication in care, and perceived barriers and facilitators to PCP-SP communication in the care of children with chronic conditions. In the spring of 2001, we surveyed all 495 New England SPs who were members of the American Academy of Pediatrics (AAP) and/or SP societies and a random sample of 495 generalist AAP members in New England. Eligible were those actively providing outpatient care. Most items were rated on a 5-point scale. RESULTS: Of those eligible, 48% (412/860) completed the questionnaire. Although 98% of respondents agreed that communication was important for good care, reported practices reflected large gaps in this area. Frequent receipt (>60% of the time) of communication about an initial referral was reported by only 28% of SPs. Barriers reported as most important involved inefficiencies in telephone contact, transcription delay, and failure to keep all providers informed when >1 specialist is involved. Important facilitators included letters or phone calls at or before the time of consultation, and clear and specific referral questions from PCPs. PCPs saw communication as more of a problem than did SPs (40% vs 28%), and reported several barriers as more important. Although 86% of respondents had access to e-mail in their practices, <20% used it often. CONCLUSIONS: PCPs and SPs sharing care for children with chronic conditions are troubled by their frequent failure or inability to contact their colleagues by phone and letter. PCPs communicate less frequently than SPs yet perceive more problems with communication. Interventions to promote efficient contact between providers at or before the time of subspecialty visits can lead to improved coordination of care, which in turn may better meet the needs of families.

Generalist-subspecialist communication about children with chronic conditions: an analysis of physician focus groups.

OBJECTIVE: To describe barriers and facilitators to effective generalist-subspecialist communication in the care of children with chronic conditions. METHODS: We conducted 5 focus groups with 14 general pediatricians and 10 pediatric specialty providers to discuss factors that facilitate or obstruct effective communication. The specialty groups included 2 nurse practitioners; the rest were pediatricians from an academic medical center and the surrounding community. We performed a content analysis to generate groups of themes and classify them as barriers or facilitators, and we returned to the participants to solicit their feedback. RESULTS: We identified 201 themes in 6 domains: the method, content, and timing of communication; system factors; provider education; and interpersonal issues. Barriers to communication mostly involved the method of communication and system factors. Most facilitating themes promoted timely communication, understanding of the reasons for referral and the nature of the child's condition, or appropriate definition of generalist and specialist roles. Participants described numerous examples where communication had direct effects on patient outcomes. Generalists and specialists agreed on many issues, although specialists discussed the pros and cons of curbside consults at length whereas generalists emphasized the importance of their own education in the referral-consultation process. CONCLUSIONS: Efforts to improve communication between pediatric generalists and specialists in the care of children with chronic conditions should emphasize the importance of timely information transfer. The content of messages is important, but lack of receipt when needed is more of a problem. Improving generalist-subspecialist communication has great potential to improve the quality of care.