Five-year outcome of children with idiopathic nephrotic syndrome: the NEPHROVIR population-based cohort study

Dossier, Claire; Delbet, Jean-Daniel; Boyer, Olivia; Daoud, Patrick; Mesples, Bettina; Pellegrino, B.; See, Hélène; Benoist, G.; Chacé, Anne; Larakeb, Anis; Hogan, Julien; Deschênes, Georges

doi:10.1007/s00467-018-4149-2

Cited by 29 publications

(19 citation statements)

References 20 publications

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“…Approximately 80% of steroid-responsive children and 70% of adult patients 178,179 undergo one or more relapses, which typically remain sensitive to steroids 180,181 . However, many patients become steroid dependent 146 or experience frequent relapses 182 (TABlE 1) after treatment discontinuation.…”

Section: Sdns and Frnsmentioning

confidence: 99%

Podocytopathies

Kopp

Anders

Suszták

et al. 2020

Nat Rev Dis Primers

305

270

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Podocytopathies are kidney diseases in which direct or indirect podocyte injury drives proteinuria or nephrotic syndrome. In children and young adults, genetic variants in >50 podocyte-expressed genes, syndromal non-podocyte-specific genes and phenocopies with other underlying genetic abnormalities cause podocytopathies associated with steroid-resistant nephrotic syndrome or severe proteinuria. A variety of genetic variants likely contribute to disease development. Among genes with non-Mendelian inheritance, variants in APOL1 have the largest effect size. In addition to genetic variants, environmental triggers such as immune-related, infection-related, toxic and haemodynamic factors and obesity are also important causes of podocyte injury and frequently combine to cause various degrees of proteinuria in children and adults. Typical manifestations on kidney biopsy are minimal change lesions and focal segmental glomerulosclerosis lesions. Standard treatment for primary podocytopathies manifesting with focal segmental glomerulosclerosis lesions includes glucocorticoids and other immunosuppressive drugs; individuals not responding with a resolution of proteinuria have a poor renal prognosis. Renin-angiotensin system antagonists help to control proteinuria and slow the progression of fibrosis. Symptomatic management may include the use of diuretics, statins, infection prophylaxis and anticoagulation. This Primer discusses a shift in paradigm from patient stratification based on kidney biopsy findings towards personalized management based on clinical, morphological and genetic data as well as pathophysiological understanding.

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Section: Sdns and Frnsmentioning

confidence: 99%

Podocytopathies

Kopp

Anders

Suszták

et al. 2020

Nat Rev Dis Primers

305

270

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“…Surprisingly, despite the patients received impressive cumulative dose of corticosteroid therapy (median 17,800 mg, maximum 35,200 mg) and/or prolonged immunosuppressive drugs, the number of patients that underwent adverse events remained limited (18; 35%), and the adverse event was severe in only two (3.8%) patients, which was far less than expected in an adult population [ 23 , 24 ]. Intravenous methylprednisolone pulses are reported to be well tolerated in childhood and are believed to be associated with a low rate of adverse events and less risk for adrenal suppression, even when delivered for years [ 25 , 26 ]. Recommended dose is 10–30 mg/kg/day for three consecutive days at monthly intervals.…”

Section: Discussionmentioning

confidence: 99%

“…Intravenous methylprednisolone pulses are reported to be well tolerated in childhood and are believed to be associated with a low rate of adverse events and less risk for adrenal suppression, even when delivered for years [25,26]. Recommended dose is 10-30 mg/kg/day for three consecutive days at monthly intervals.…”

Section: Unexpected Tolerance Of Intensive and Prolonged Treatmentmentioning

confidence: 99%

Child–Adult Transition in Sarcoidosis: A Series of 52 Patients

Chauveau

Jeny

Montagne

et al. 2020

JCM

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(1) Background: Pediatric sarcoidosis is a rare and mostly severe disease. Very few pediatric series with a prolonged follow-up are reported. We aimed to evaluate the evolution of pediatric sarcoidosis in adulthood. (2) Material and methods: Patients over 18-years-old with a pediatric-onset sarcoidosis (≤15-year-old) who completed at least a three-year follow-up in French expert centers were included. Clinical information at presentation and outcome in adulthood were studied. (3) Results: A total of 52 patients were included (34 prospectively in childhood and 18 retrospectively in adulthood), with a mean age of 12 (±2.7) at diagnosis. The median duration time of follow-up was 11.5 years (range 3–44.5). Relapses mostly occurred during treatment decrease (84.5%), others within the three years after treatment interruption (9.1%), and rarely when the disease was stable for more than three years (6.4%). Sarcoidosis was severe in 11 (21.2%) in adulthood. Patients received a high corticosteroid cumulative dose (median 17,900 mg) for a median duration of five years (range 0–32), resulting in mostly mild (18; 35.3%) and rarely severe (2; 3.8%) adverse events. (4) Conclusions: Pediatric-onset sarcoidosis needed a long-term treatment in almost half of the patients. Around one fifth of pediatric-onset sarcoidosis patients had severe sarcoidosis consequences in adulthood.

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“…Although the majority of children with idiopathic nephrotic syndrome (INS) are steroid sensitive (SSNS), 80% of them will experience relapses and a chronic evolution of the disease [ 1 ]. A significant proportion of frequent relapsers (FRNS) and steroid dependent (SDNS) will have a protracted course exceeding 10 years and be exposed to oral prednisone and immunosuppressive drugs beyond adult age [ 2 ].…”

Section: Introductionmentioning

confidence: 99%

A global antiB cell strategy combining obinutuzumab and daratumumab in severe pediatric nephrotic syndrome

et al. 2020

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Background Steroid-sensitive nephrotic syndrome (SSNS) is, in most patients, a chronic disease with 80% experiencing at least one relapse after first flare. B cell depletion using rituximab is effective in preventing relapse in steroid-dependent (SDNS) patients but fails to maintain long-term remission following B cell recovery, possibly due to development of autoreactive long-lived plasma cells. We investigated sequential combination of antiCD20 antibody targeting all B cell subsets, and antiCD38 antibody with high plasma cell cytotoxicity in patients with uncontrolled SDNS after failure of one or several attempts at B cell depletion. Methods Fourteen patients with median disease duration 7.8 years received 1000 mg/1.73 m 2 obinutuzumab followed by 1000 mg/1.73 m 2 daratumumab 2 weeks later. Oral immunosuppression was discontinued within 6 weeks, and biological monitoring performed monthly until B cell recovery. Results Median age at treatment was 11.0 [IQR 10.4–14.4] years. B cell depletion was achieved in all patients, and B cell reconstitution occurred in all at median 9.5 months after obinutuzumab injection. After median follow-up 20.3 months (IQR 11.5–22.6), 5/14 patients relapsed including 4 within 100 days following B cell repletion. Relapse-free survival was 60% at 24 months from obinutuzumab infusion. Mild infusion reactions were reported in 3/14 patients during obinutuzumab and 4/14 during daratumumab infusions. Mild transient neutropenia (500–1000/mm 3 ) occurred in 2/14 patients. Intravenous immunoglobulins were given to 12/14 patients due to hypogammaglobulinemia. Low IgA and IgM levels were noted in 8 and 14 patients, respectively. No severe infection was reported. Conclusion Global antiB cell strategy combining obinutuzumab and daratumumab induces prolonged peripheral B cell depletion and remission in children with difficult-to-treat SDNS. Electronic supplementary material The online version of this article (10.1007/s00467-020-04811-0) contains supplementary material, which is available to authorized users.

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Five-year outcome of children with idiopathic nephrotic syndrome: the NEPHROVIR population-based cohort study

Cited by 29 publications

References 20 publications

Podocytopathies

Podocytopathies

Child–Adult Transition in Sarcoidosis: A Series of 52 Patients

A global antiB cell strategy combining obinutuzumab and daratumumab in severe pediatric nephrotic syndrome

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