|
| Reference | Patient | 2CdA regimen | Outcome | Toxicities |
|
| Khezri 2011 [12] | Series of 8 patients: All with diffuse skin involvement; 1 patient with additional pituitary and airway involvement; 1 patient with pituitary, adenoid, and soft palate involvement | 5 out of 8 patients received treatment: 0.14 mg/kg/day x 5 days every 4 weeks x 5–8 cycles | All patients had substantial improvement in established lesions; no new lesions in follow-up period (3 months to 8 years) | Joint pain and night sweats in 1 patient; no serious adverse effects reported |
|
| Gupta 2016 [21] | 23yo M with diffuse skin involvement and DI | 0.14 mg/kg/day x 5 days every 1 month x 8 cycles | Stopped developing lesions after cycle 2; marked improvement after cycle 5 | None reported |
|
| Adisen 2017 [22] | 19yo F with diffuse skin involvement | 0.14 mg/kg/day x 5 days every 1 month x 7 cycles | Resolution of many lesions; residual hyperpigmented macules remaining; no new lesions in 2-year follow-up | None reported |
|
| Briones 2018 [23] | 55yo M with diffuse skin and laryngopharyngeal involvement | Presumed 0.14 mg/kg/day for 5 days, every 1 month X 3 cycles (6-8 cycles planned, stopped due to thrombocytopenia) | Clinical resolution of changes in voice, significant improvement in skin, lip, and gingiva; complete resolution of all lesions and no new lesions at 10 months follow-u | Thrombocytopenia (early termination of planned therapy) |
|
| Patra 2019 [24] | 9yo M with diffuse skin, DI, optic atrophy, and multiple intracranial lesions | 0.14 mg/kg/day x 5 days every 1 month x 6 cycles | 30–40% flattening of skin lesions, decrease in intracranial lesion size, and improvement in diabetes insipidus and vision | Asymptomatic transient leukopenia |
|
| Tuan 2019 [25] | 3 patients all with diffuse skin involvement and DI; 1 patient with respiratory involvement requirement tracheostomy; 1 patient with bulbar involvement | 0.14 mg/kg/day x 5 days every 1 month x 5–9 cycles | Significant resolution of cutaneous and mucosal lesions; no signs of relapse after 52–66 months of follow-up | None reported |
|
| Al-Tarcheh 2020 [26] | 24yo F with diffuse skin, DI, tracheal, knee joint, and nasopharynx involvement | 0.14 mg/kg/day x 5 days every 1 month x 6 cycles | Resolution of nasopharynx mass and tracheal lesion; no new skin lesions but no regression of established lesions | None reported |
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