Sustained response to intrathecal rituximab in EBV associated Post-transplant lymphoproliferative disease confined to the central nervous system following haematopoietic stem cell transplant.

Post-transplant lymphoproliferative disorder (PTLD) in the central nervous system (CNS) is a very rare complication of haematopoietic stem cell transplant (HSCT) and has a dismal prognosis. We report the successful treatment of this disorder with intrathecal rituximab therapy in two children who developed isolated CNS PTLD after HSCT. These children had failed to respond to standard chemotherapy, intravenous rituximab and EBV specific cellular therapy.

Successful allogeneic bone marrow transplant for Niemann-Pick disease type C2 is likely to be associated with a severe 'graft versus substrate' effect.

Niemann-Pick disease type C2 (NPC2) is caused by the inherited deficiency of a lysosomal cholesterol transport protein, NPC2 protein. Many cases of NPC2 present in early infancy with inflammatory lung disease, with subsequent severe neurological disease and death in early childhood. This disease is theoretically correctable by bone marrow transplantation (BMT), as the NPC2 protein is small and soluble and secreted and recaptured by the mannose-6-phosphate pathway.

Congenital diverticulum of the bladder mimicking tumour.

The incidence of bladder diverticula is low and identification is usually straightforward with contrast studies. We present a case of a child who had a clearly identified bladder diverticulum as well as a soft-tissue lesion in the posterior wall of the bladder that was suspected to be a rhabdomyosarcoma. Despite ultrasound scanning, micturating cystourethrogram, computed tomography and cystoscopy, open surgery was required to rule out tumour.

Acute renal papillary necrosis with complete bilateral ureteral obstruction in a child.

A 9-year-old girl presented with apparent meningococcal septicemia and developed acute renal failure after 48 hours of treatment with antibiotics and analgesics. Early ultrasound scanning demonstrated mild bilateral hydronephrosis and hydroureter. Intravenous urography showed slow contrast uptake with delay nephrogram and no contrast entering the bladder.