Sirolimus
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Sirolimus Avascular femoral osteonecrosis following compassionate use: case report.
A 21-year-old man developed avascular femoral osteonecrosis during compassionate use of sirolimus for blue rubber bleb nevus syndrome (BRBNS). The man, was diagnosed with BRBNS at the age of 5 years. He had a history of multiple vascular lesions in liver, gastrointestinal (GI) tract and skin. In childhood, he underwent resection of bowel due to intussusception caused by intestinal lesions. His anaemia was maintained with iron supplements, but GI blood loss deteriorated in 2011 requiring several transfusions. Capsule endoscopy revealed >100 lesions along small intestine. He was started on octreotide and ferric carboxymaltose which showed good haematologic response. In August 2014, he developed an avascular left hip osteonecrosis requiring temporarily discontinuation of octreotide and starting weekly transfusions. Concomitantly, from 2012 to 2016, he underwent several interventions including endoscopic sclerosis, argon plasma photocoagulation, endoscopic ligature with endoloop and treatment with polidocanol, but no improvement was noted. After 5 years of octreotide treatment, in 2017, he required blood transfusions. Therefore, a compassionate-use of sirolimus [rapamycin] was considered. He was closely monitored due to history of hip avascular osteonecrosis and started on sirolimus 0.07 mg/kg/d (5 mg/day) [route not stated]. After 5 weeks of initiation of sirolimus, his haemoglobin increased from 8.5 to 12 g/dL. The dose of reduced to 3.5 mg/d. After 6 months, he developed contralateral hip discomfort and MRI revealed avascular femoral osteonecrosis. The man underwent surgery with stem cell injection and achieved an adequate clinical improvement. After 2 years of sirolimus initiation, he remained with an excellent control of anaemia and GI haemorrhage without side effects. Fernandez Gil M, et al. Successful management of anemia with sirolimus in blue rubber bleb nevus syndrome: case report and update. [Review]. Revista Espanola de Enfermedades Digestivas 111: 643-647, No. 8, Aug 2019. Available from: URL: http://doi.org/10.17235/reed.2019.6250/2019
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Reactions 5 Sep 2020 No. 1820
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