Translocations involving the 8q24 MYC locus more frequently manifest as t(6;8)(p21;q24), and, given its association with specific clinicopathological features suggesting even more aggressive behaviour, t(6;8)(p21;q24) indicate a genetically defined subgroup within BPDCN.

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Background and Objectives: SHSPN leads to ESRD in >10% of children, yet no consensus exists on its treatment. We present 32 such cases treated at our center over 12 years. We compare and analyse the clinical outcomes of a prolonged PMP regimen with added immunosuppression and adjuvant therapies, to a regimen of POS with the same additional therapies. Design, Setting, Participants & Measurements:This case series defines SHSPN as AKI, or nephrotic range or rapidly rising proteinuria. The PMP dosing is 30 mg/kg/day (up to 1gr) for three doses, and then weekly until weaned off based on clinical response. The POS dose is 2 mg/kg/day (up to 60 mg), tapered over 3-6 months based on clinical response.The Supplemental Immunosuppression Includes: oral steroids at 2 mg/kg/day (up to 60 mg), tapered to alternate day by 4-6 weeks and mycophenolate at 300-600 mg/m2/dose for the PMP group; and oral cyclophosphamide at 1.5-2 mg/kg/day for 10-12 weeks, or a purine synthesis inhibitor mainly azathioprine 2mg/kg/day for the POS group. Adjuvant therapies were identical for the two groups: angiotensin converting enzyme inhibitors and/or angiotensin receptor blockers and either vitamin E, or omega-3 fatty acids.Results: 23 patients received PMP and 9 POS; there were no differences at presentation. Mean follow up is 5 years (1.5-12.5 years); one progression to ESRD occured. After 2 years PMP demonstrates significant and beneficial improvements in GFR (mean e GFR 96.33 mL/min/1.73 m 2 vs 118.5 mL/min/1.73 m 2 p= 0.02), and mean arterial pressure (84.7mmHg vs 77.1 mmHg p=0.015) as well as proteinuria control (mean UpUc 5.25 mg/mg vs 0.41 mg/mg p= 0.008). Additionally, there are no differences between the two treatments with regards height and BMI Z scores.Conclusions: PMP, with adjuvant immunosuppressive and nephro-protective measures offer a significant reduction of proteinuria, and MAP plus improved GFR with minimal morbidity to children with SHSPN.