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Abstract: TH-PO519

New Treatments, New Challenges: Nephrotoxicity-Associated Naxitamab in Pediatric Patients with High-Risk Neuroblastoma

Session Information

  • Pediatric Nephrology - I
    November 02, 2023 | Location: Exhibit Hall, Pennsylvania Convention Center
    Abstract Time: 10:00 AM - 12:00 PM

Category: Pediatric Nephrology

  • 1900 Pediatric Nephrology


  • Madrid Aris, Alvaro, Hospital Sant Joan de Deu, Barcelona, Catalunya, Spain
  • Arango Sancho, Pedro, Hospital Sant Joan de Deu, Barcelona, Catalunya, Spain

Neuroblastoma is the most common extracranial solid tumor in pediatrics,having a poor survival in high- risk (HR) tumors. Naxitamab(hu3F8) is a humanized monoclonal antibody anti-dysialoganglioside (GD2) approved for treatment of > 1 year-old and adults with refractory/relapsed HR-neuroblastoma limited to bone or bone marrow. Our hospital has been the first center (2017) worldwide use it (clinical trials/compassionate use), obtaining excellent results.


Retrospective descriptive study including 244 patients (41% female-101-and 59% male-143-) using Naxitamab (monotherapy and/or associated with chemotherapy) from June 2017 to current day (6 years) in whom renal involvement and/or hypertension (HT) was evaluated.


Mean age was 8 years, presenting nephrotoxicity of some type up to 26.6%(65):HT(11.9%/29),acute renal damage(ARD10.2%/25)and proteinuria(5.3%/13),developing all of them during the infusion or the first 3 cycles.In case of HT,only in 6 patients an ABPM was performed,observing:2 nocturnal-HT,2 diurnal-HT without specific-pattern and 2 disautonomic-pattern,not previously observed.Among the ARD,all cases were tubular except for one patient who presented clinical-analytical pattern of acute tubule- interstitial nephritis(AIN).Eight of them(32%)presented possible confounding factors in the development of(previous chemotherapy,ibuprofen or radiotherapy).Among patients with proteinuria(none nephrotic range):38% tubular,38% glomerular and 23% mixed.2 patients presented ARD+AHT and 3 a combination of AHT+ARD+proteinuria.Of these last,all of them received prior chemotherapy,leaving 2 of them with chronic renal damage(CKD stage 2 and 3).67 patients(27.4%)died due to progression of their underlying disease.


Management of HR-neuroblastoma remains a daily challenge. Naxitamab is an emerging therapy in this type of tumors, although there are few studies describing its AE.Previous studies of our group in mice explain the involvement of the myelin sheaths of the autonomic nervous system with this drug, which could explain, among others, the dysautonomic pattern of blood pressure presented. Short- and long-term follow-up, the systematic performance of ABPM and the use of early markers of renal damage, could lead to a more efficient management of complications derived from this new treatment.