ABSTRACT Introduction: Rituximab (RTX) is a therapeutic option in pediatric difficult-to-treat idiopathic nephrotic syndrome (NS). We aimed to assess the efficacy and safety of RTX use in these patients. Method: A retrospective study of all patients with idiopathic NS treated with RTX was conducted in a pediatric nephrology division of a tertiary hospital. Demographic, anthropometric, clinical and analytical data were collected prior to treatment and at 6, 12, and 24 months. Results: Sixteen patients were included (11 males), with a median (25th–75th percentile, P25–P75) age at diagnosis of 2 (2.0–2.8) years. Fifteen were steroid-sensitive and 1 was steroid-resistant and sensitive to cyclosporine. The median age at administration of RTX was 10 (6.3–14.0) years. Throughout a median follow-up time of 2.5 (1.0–3.0) years, 6 (37.5%) patients achieved partial remission and 7 (43.8%) had no relapses and were not taking any immunosuppressants at the 24-month follow-up visit. Regarding complications, 1 patient presented persistent hypogammaglobulinemia. Compared with the 12-month period before RTX, there was a decrease in the median number of relapses at 6 and 12 months [3 (3.0–4.0) vs 0 (0–0.8) and 0.50 (0–1.0), respectively; p = 0.001] and in the daily steroids dose (mg/kg/day) at 6, 12, and 24 months [0.29 (0.15–0.67)vs [0.10 (0.07–0.13); p = 0.001], [0.12 (0.05–0.22); p = 0.005] and [0.07(0.04–0.18); p = 0.021]], respectively. There was also a reduction in the median BMI z score at 24 months [2.11 (0.45–3.70) vs. 2.93 (2.01–3.98); p = 0.049]. Conclusion: Our results confirm the efficacy and safety of RTX use in pediatric idiopathic NS and highlight its’ potential cardiometabolic benefits. Introduction (RTX difficulttotreat difficult treat NS. . (NS) Method hospital Demographic anthropometric Results 11 (1 males, males , males) 25th–75th 25th75th thth 25th 75th th percentile P25–P75 P25P75 PP P25 P75 P 2.0–2.8 2028 8 (2.0–2.8 years steroidsensitive steroid steroidresistant resistant cyclosporine 6.3–14.0 63140 3 14 (6.3–14.0 followup follow up 25 5 2. 1.0–3.0 1030 (1.0–3.0 37.5% 375 37 (37.5% 43.8% 438 43 (43.8% 24month month visit complications hypogammaglobulinemia 12month [ 3.0–4.0 3040 4 (3.0–4.0 0–0.8 008 (0–0.8 050 50 0.5 0–1.0, 010 0–1.0 (0–1.0) respectively 0.001 0001 001 mg/kg/day mgkgday mg kg day (mg/kg/day 0.29 029 29 [0.2 0.15–0.67vs 015067vs 0.15–0.67 15 67 0.10 [0.1 0.07–0.13 007013 07 13 (0.07–0.13) 0.001, 0.12 012 0.05–0.22 005022 05 22 (0.05–0.22) 0.005 0005 005 0.070.04–0.18 007004018 0.07 0.04–0.18 04 18 [0.07(0.04–0.18) 0.021, 0021 0.021 021 0.021]] 2.11 211 [2.1 0.45–3.70 045370 45 70 (0.45–3.70 293 93 2.9 2.01–3.98 201398 01 98 (2.01–3.98) 0.049. 0049 0.049 049 0.049] Conclusion its benefits (NS ( P25–P7 P25P7 P2 P7 2.0–2. 202 (2.0–2. 6.3–14. 6314 (6.3–14. 1.0–3. 103 (1.0–3. 37.5 (37.5 43.8 (43.8 3.0–4. 304 (3.0–4. 0–0. 00 (0–0. 0. 0–1. (0–1.0 0.00 000 0.2 02 [0. 67vs 015067 0.15–0.6 0.1 0.07–0.1 00701 (0.07–0.13 0.05–0.2 00502 (0.05–0.22 070 0.070.04–0.1 00700401 007 0.0 004018 0.04–0.1 [0.07(0.04–0.18 002 0.02 0.021] 2.1 21 [2. 0.45–3.7 04537 (0.45–3.7 9 2.01–3.9 20139 (2.01–3.98 004 0.04 P25–P P25P 2.0–2 20 (2.0–2 6.3–14 631 (6.3–14 1.0–3 (1.0–3 37. (37. 43. (43. 3.0–4 30 (3.0–4 0–0 (0–0 0–1 (0–1. [0 01506 0.15–0. 0.07–0. 0070 (0.07–0.1 0.05–0. 0050 (0.05–0.2 0.070.04–0. 0070040 00401 0.04–0. [0.07(0.04–0.1 [2 0.45–3. 0453 (0.45–3. 2.01–3. 2013 (2.01–3.9 2.0– (2.0– 6.3–1 63 (6.3–1 1.0– (1.0– (37 (43 3.0– (3.0– 0– (0– (0–1 0150 0.15–0 0.07–0 (0.07–0. 0.05–0 (0.05–0. 0.070.04–0 007004 0040 0.04–0 [0.07(0.04–0. 0.45–3 045 (0.45–3 2.01–3 201 (2.01–3. 2.0 (2.0 6.3– (6.3– 1.0 (1.0 (3 (4 3.0 (3.0 (0 015 0.15– 0.07– (0.07–0 0.05– (0.05–0 0.070.04– 00700 0.04– [0.07(0.04–0 0.45– (0.45– 2.01– (2.01–3 (2. 6.3 (6.3 1. (1. 3. (3. 0.15 (0.07– 0.05 (0.05– 0.070.04 [0.07(0.04– 0.45 (0.45 2.01 (2.01– (2 6. (6. (0.07 (0.05 0.070.0 [0.07(0.04 0.4 (0.4 (2.01 (6 (0.0 0.070. [0.07(0.0 (0. 0.070 [0.07(0. [0.07(0 [0.07( [0.07 [0.0
Resumo Introdução: Rituximabe (RTX) é uma opção terapêutica na síndrome nefrótica (SN) idiopática pediátrica de difícil tratamento. Visamos avaliar eficácia e segurança do uso de RTX nestes pacientes. Método: Realizou-se estudo retrospectivo de todos os pacientes com SN idiopática tratados com RTX, em uma unidade de nefrologia pediátrica de um hospital terciário. Dados demográficos, antropométricos, clínicos e analíticos foram coletados antes do tratamento e aos 6, 12 e 24 meses. Resultados: Incluímos 16 pacientes (11 do sexo masculino), com idade mediana (percentil 25–75, P25–P75) de 2 (2,0–2,8) anos ao diagnóstico. Quinze eram sensíveis a esteroides, e 1 resistente a esteroides e sensível à ciclosporina.A idade mediana na administração do RTX foi 10 (6,3–14,0) anos. Durante um tempo mediano de acompanhamento de 2,5(1,0–3,0) anos, 6 (37,5%) pacientes alcançaram remissão parcial e 7 (43,8%) não tiveram recidivas e não estavam tomando imunossupressor no acompanhamento aos 24 meses. Quanto às complicações,1 paciente apresentou hipogamaglobulinemia persistente. Comparado ao período de12 meses anterior ao RTX, houve diminuição no número mediano de recidivas em 6 e 12 meses [3 (3,0–4,0) vs 0 (0–0,8) e 0,50 (0–1,0), respectivamente; p = 0,001] e na dose diária de esteroides (mg/kg/dia) aos 6, 12 e 24 meses [0,29 (0,15–0,67) >vs [0,10 (0,07–0,13); p = 0,001], [0,12 (0,05–0,22); p = 0,005] e [0,07 (0,04–0,18); p = 0,021], respectivamente. Houve também redução na mediana do escore z do IMC aos 24 meses [2,11 (0,45–3,70) vs 2,93 (2,01–3,98);p = 0,049]. Conclusões: Nossos resultados confirmam a eficácia e segurança do uso de RTX em SN idiopática pediátrica, destacando seus potenciais benefícios cardiometabólicos. Introdução (RTX (SN Método Realizouse Realizou se terciário demográficos antropométricos Resultados 11 (1 masculino, masculino , masculino) percentil 2575 25 75 25–75 P25–P75 P25P75 PP P25 P75 P 2,0–2,8 2028 8 (2,0–2,8 diagnóstico ciclosporinaA ciclosporina A 6,3–14,0 63140 3 14 (6,3–14,0 2,51,0–3,0 251030 2,5 1,0–3,0 5 2,5(1,0–3,0 37,5% 375 37 (37,5% 43,8% 438 43 (43,8% complicações1 complicações complicações, persistente de1 [ 3,0–4,0 3040 4 (3,0–4,0 0–0,8 008 (0–0,8 050 50 0,5 0–1,0, 010 0–1,0 (0–1,0) respectivamente 0,001 0001 001 mg/kg/dia mgkgdia mg kg dia (mg/kg/dia 0,29 029 29 [0,2 0,15–0,67 015067 15 67 (0,15–0,67 0,10 [0,1 0,07–0,13 007013 07 13 (0,07–0,13) 0,001, 0,12 012 0,05–0,22 005022 05 22 (0,05–0,22) 0,005 0005 005 0,07 007 [0,0 0,04–0,18 004018 04 18 (0,04–0,18) 0,021, 0021 0,021 021 0,021] 2,11 211 [2,1 0,45–3,70 045370 45 70 (0,45–3,70 293 93 2,9 2,01–3,98p 201398p 2,01–3,98 01 98 0,049. 0049 0,049 . 049 0,049] Conclusões cardiometabólicos ( 257 25–7 P25–P7 P25P7 P2 P7 2,0–2, 202 (2,0–2, 6,3–14, 6314 (6,3–14, 51 2,51,0–3, 25103 2, 1030 1,0–3, 2,5(1,0–3, 37,5 (37,5 43,8 (43,8 3,0–4, 304 (3,0–4, 0–0, 00 (0–0, 0, 0–1, (0–1,0 0,00 000 0,2 02 [0, 0,15–0,6 01506 (0,15–0,6 0,1 0,07–0,1 00701 (0,07–0,13 0,05–0,2 00502 (0,05–0,22 0,0 0,04–0,1 00401 (0,04–0,18 002 0,02 2,1 21 [2, 0,45–3,7 04537 (0,45–3,7 9 98p 201398 2,01–3,9 004 0,04 25– P25–P P25P 2,0–2 20 (2,0–2 6,3–14 631 (6,3–14 2,51,0–3 2510 103 1,0–3 2,5(1,0–3 37, (37, 43, (43, 3,0–4 30 (3,0–4 0–0 (0–0 0–1 (0–1, [0 0,15–0, 0150 (0,15–0, 0,07–0, 0070 (0,07–0,1 0,05–0, 0050 (0,05–0,2 0,04–0, 0040 (0,04–0,1 [2 0,45–3, 0453 (0,45–3, 20139 2,01–3, 2,0– (2,0– 6,3–1 63 (6,3–1 2,51,0– 251 1,0– 2,5(1,0– (37 (43 3,0– (3,0– 0– (0– (0–1 0,15–0 015 (0,15–0 0,07–0 (0,07–0, 0,05–0 (0,05–0, 0,04–0 (0,04–0, 0,45–3 045 (0,45–3 2013 2,01–3 2,0 (2,0 6,3– (6,3– 2,51,0 1,0 2,5(1,0 (3 (4 3,0 (3,0 (0 0,15– (0,15– 0,07– (0,07–0 0,05– (0,05–0 0,04– (0,04–0 0,45– (0,45– 201 2,01– (2, 6,3 (6,3 2,51, 1, 2,5(1, 3, (3, 0,15 (0,15 (0,07– 0,05 (0,05– (0,04– 0,45 (0,45 2,01 (2 (6, 2,51 2,5(1 (0,1 (0,07 (0,05 (0,04 0,4 (0,4 (6 2,5( (0, (0,0