Table 3.

Efficacy and safety of tofacitinib in the treatment of patients with interferonopathies

Study ID (reference)	Disease (no. of study patients)a	Sex/age at enrollment/age at onset	Previous treatmentb	Mutation	Dosage/duration of treatment/association with steroids	Efficacy/skin and/or systemic improvement/efficacy comments	Safety
4 [27]	CANDLE (1)	Female 18 years NA	GLC, CyP, AZA, CFM, HCQ, IVIG, ANK, IFX, ABA, HpT	Not confirmed	15–20 mg/24 h NA NA	Yes NA Lipodystrophy, chilblains, and hair loss improvement	Increase in creatine kinase and low-density lipoprotein cholesterol levels
5 [28]	FCh-L(SM) (1)	Female 60 years 3 years	NA	TMEM173 G166E	10 mg/24 h 17 days No	Yes Yes Discomfort and pain on fingers improvement	NA
	FCh-L(SM) (2)	Female 58 years 2 months	NA	TMEM173 G166E	10 mg/24 h 17 days No	Yes Yes Discomfort and pain on fingers improvement	NA
9 [29]	SAVI (1)	Female 6 years 1 year	NA	Not confirmed	7.5 mg/24 h 12 months Yes	Yes Yes Pulmonary, rash, arthralgias and analytical improvement	NA
13 [30]	SAVI (1)	Male 9 years 6 months	NA	De novo double-mutant TMEM173 (S102P-F279L)	5 mg/day 3 months NA	Yes NA Pulmonary defect unchanged	NA
14 [31]	SAVI (1)	Female 12 months 3 months	GLC + IVIG	De novo TMEM173 V155M	5 mg/day 10 months Yes	Yes Partially Rash improvement, but pulmonary involvement worsening and fever	Died due to acute respiratory failure
	SAVI (2)	Male 60 months 54 months	GLC + IVIG, CFM	De novo TMEM173 V155M	5 mg/24 h 10 months Yes	Yes Partially Rash improvement while pulmonary involvement worsening	No adverse events

ABA abatacept, ANK anakinra CFM cyclophosphamide, CyP cyclosporine, HpT Hyperbaric therapy,NA not available

^aFCh-L(SM) Familial Chilblain Lupus due to a gain-of-function mutation in STING

^bPrevious treatment with JAK/STAT pathway inhibitors is underlined