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Table 1 Reported cases of GSD treated with sirolimus (rapamycin)

From: Gorham-Stout disease successfully treated with sirolimus (rapamycin): a case report and review of the literature

Author Gender Agea Nationality Therapy Sirolimus Dose Manifestation Follow up after treatment Outcome
Cramer et al. [5] male 18 yrs U.S Sirolimus zoledronic acid thoracoscopic surgery Titrated to 9-12 mg/L, twice daily intractable pleural effusion, and multiple rib fractures 18 mos effusion improved and no further skeletal fracture
Araujo et al. [6] male 5 yrs Brazil Sirolimus
1 mg/twice daily ribs and sternum lesions and haemothorax 8 mos pulmonary parenchyma
normal and good bone remodelling
Garcia et al. [7] female 43 yrs Spain Sirolimus
Titrated to 4-10 mg/L, twice daily pleural effusion, abdominal mass, rib 2 mos symptoms vanish
Mohammad et al. [8] male 12 yrs Malaysia Sirolimus NA right chylothorax, right clavicle, humerus and ulna osteolysis NA responded well
Mohammad et al. [8] male 8 yrs Malaysia Sirolimus NA cervical and thoracic spine lytic bone lesion and massive left pleural effusion 3 mos responded poorly
Colin et al. [9] female 3 yrs Mexico Sirolimus 0.05 mg/kg/d right femur, skull osteolysis, left femur pathologic fracture NA control of the disease with no pain
Cakir et al. [10] female 2 yrs Turkey Sirolimus NA right sided pleural effusion, ribs and thoracic and lumbar vertebral lytic lesions 14 mos symptoms and signs vanish
Wei et al. [11] female 17 yrs U.S Sirolimus NA right femur pathological fracture 7 mos symptoms and signs vanish
Hall et al. [12] female 10 yrs U.S Sirolimus NA right 9th rib osteolysis 24 mos controlled
Gordon et al. [13] female 13 yrs Britain Sirolimus Titrated to 10-15 mg/L, twice daily multiple vertebral body and pelvic osteolysis NA chylous leakage ceased, pain remission, and no further bone destruction
Mo et al. [14] male 14 yrs U.S Sirolimus Titrated to 7–13 mg/L, twice daily thoracic spine and rib osteolysis 26 mos symptoms and signs vanish
Wang et al. [3] female 3 yrs China Sirolimus Titrated to 10-15 mg/L, twice daily humerus and scapula osteolysis and resorption in clavicle. right-sided pleural effusion 13 mos The mass shrank and discolored skin lessened
Nozawa et al. [15] male 27 yrs Japan Sirolimus+
2 mg/day extraosseous soft-tissue mass; multiple osteolysis 6 mos responded poorly
Suero et al. [16] male 26 yrs Germany Sirolimus+
thoracoscopic pleurodesis
NA compression fracture of the vertebrae and pleural effusion 48 mos Recurrence (30 months after withdrawal)
Cho et al. [17] female 11 yrs Korea Sirolimus+
Titrated to 9-12 mg/L, twice daily right clavicle and 1st rib osteolytic cortical resorption and chyle leakage 24 mos symptoms are well-controlled
Present case male 1 yr China Sirolimus+
thoracoscopic surgery
Titrated to 7–13 mg/L, twice daily clavicle resorption. right-sided pleural effusion and right femur fracture 26 mos symptoms and signs vanish
  1. a Age at initiation of sirolimus treatment