Background Scleroderma is a connective tissue disorder that is associated with changes in the skin and sometimes in internal organs. Digital ulcers in these patients can be difficult to treat and are associated with significant morbidity. Published literature shows that bosentan, an oral endothelin receptor antagonist has been useful in the treatment of digital ulcers in adults with scleroderma. However there has been no such literature published on paediatric patients.
Objectives To evaluate the clinical response of three patients with digital ulcers treated with bosentan.
Methods We present our experience of treating three children with systemic sclerosis and digital ulcers with bosentan.
Case1: 12 year old female patient with diagnosis of systemic sclerosis/juvenile dermatomyositis overlap presented with Raynaud’s phenomenon, heliotrope rash, digital ulcers, tightening around mouth, exertional dyspnoea, rash over knuckles and nail fold capillary dilatation. Her bloods revealed she was ANA positive 1:2560 speckled pattern and ENA and RF positive. She had abnormal capillaries, oesophageal dysmotility and interstitial lung disease.
Case 2: 10 year old female patient presented with linear skin lesions on thigh, exertional dyspnoea, dysphagia, digital ulcers and Raynaud’s phenomena. Her bloods revealed she was ANA positive 1:2560 homogenous pattern, ENA and AntiSCL antibody positive. She had an abnormal capillaroscopy, oesophageal dysmotility and interstitial lung disease.
Case 3: 8 year old female patient had diffuse cutaneous skin disease, severe digital ulcers, hypopigmeted rash on knuckles, abnormal nail fold capillary dilatation and hypertension on presentation. Her bloods revealed she was ANA positive 1:2560 and anti U3RNP antibody positive. She had abnormal capillaries and oesophageal dysmotility but no interstitial lung disease.
2 patients were treated with bosentan at dose of 125 mg twice daily and one patient received between 125 mg to 250 mg twice daily. All the three patients were treated with nifedipine and glyceryl trinitrate patches for Raynaud’s phenomenon. They received systemic treatment including steroids and methotrexate, two of these patients received cyclophosphamide, one patient received mycophenolate mofetil and one patient received infliximab and subsequently adalimumab in addition. The third patient described had severe digital ulcers requiring iloprost/epoprostinol initially.
Results Case 1 (scleroderma/juvenile dermatomyositis overlap) showed good response to bosentan. She had fewer new ulcers and her digital ulcers completely resolved. In this case bosentan was stopped after 4 years. Case 2 and 3 (systemic sclerosis) have been on bosentan for 18 months and 6 months respectively. Ulcers present at the time of diagnosis improved and there were fewer new ulcers in these patients. None of the three patients had side effects related to bosentan.
Conclusions In our experience we have found bosentan to be safe and useful in the treatment of digital ulcers in these three children with scleroderma.
Disclosure of Interest None Declared
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