Table 2.
Individual descriptions of the 27 female pations.
| Patient number | Age at start (years) | Target organs | Sirolimus exposure (months) | Daily dose (mg) of sirolimus | Combining DMARDs | Cause of cessation | Number of DMARDs ahead of sirolimus | SDI at start | SDI at last follow-up on sirolimus |
|---|---|---|---|---|---|---|---|---|---|
| 1 | 58 | Musculoskeletal, leukocytopenia | 117 | 2 | HQ | Treatment ongoing | 2 | 1 | 1 |
| 2 | 65 | Musculoskeletal, discoid lupus | 10 | 3 | None | Rash, swelling of legs | 3 | 6 | 6 |
| 3 | 43 | Musculoskeletal | 18 | 1 | HQ, MMF | Decreasing effect | 6 | 3 | 3 |
| 4 | 53 | Musculoskeletal | 96 | 1 | None | Treatment ongoing | 4 | 0 | 1 |
| 5 | 62 | Musculoskeletal | 35 | 2 | HQ, MTX | Itching, headache | 5 | 1 | 3 |
| 6∗† | 51 | Musculoskeletal, leukocytopenia | 3 | 1 | None | Infection, lack of efficacy | 6 | 2 | 2 |
| 7 | 59 | Musculoskeletal, malar rash | 13 | 1 | HQ | Increased liver enzymes | 3 | 0 | 1 |
| 8 | 37 | Musculoskeletal, alopecia, pleuritis | 4 | 2 | None | Nausea | 5 | 1 | 1 |
| 9 | 27 | Musculoskeletal | 129 | 1 | HQ | Treatment ongoing | 4 | 0 | 2 |
| 10† | 56 | Musculoskeletal | 31 | 2 | None | Malignancy | 3 | 1 | 1 |
| 11 | 35 | Musculoskeletal | 7 | 1 | None | Itching, fatigue | 2 | 1 | 1 |
| 12 | 61 | Musculoskeletal | 63 | 2 | HQ | Treatment ongoing | 2 | 1 | 2 |
| 13 | 44 | Musculoskeletal | 140 | 3 | HQ, MMF | Treatment ongoing | 4 | 1 | 1 |
| 14 | 52 | Musculoskeletal | 111 | 2 | None | Treatment ongoing | 3 | 1 | 2 |
| 15† | 50 | Musculoskeletal, malar rash | 127 | 2 | None | Infections | 5 | 2 | 7 |
| 16∗ | 32 | Musculoskeletal | 2 | 1 | HQ | Nausea | 3 | 0 | 0 |
| 17 | 38 | Musculoskeletal, photosensitivity | 4 | 1 | HQ | Lack of efficacy | 4 | 0 | 0 |
| 18∗ | 48 | Musculoskeletal | 3 | 1 | HQ | Nausea | 2 | 0 | 0 |
| 19 | 20 | Musculoskeletal | 70 | 1 | HQ | Reached remission | 4 | 0 | 0 |
| 20 | 50 | Musculoskeletal | 104 | 1 | HQ | Angioedema | 4 | 0 | 1 |
| 21 | 48 | Musculoskeletal | 7 | 1 | HQ, MMF | Nausea | 3 | 1 | 1 |
| 22 | 21 | Lupus profundus | 27 | 1 | HQ | Diarrhea | 6 | 1 | 1 |
| 23∗ | 38 | Musculoskeletal, malar rash | 3 | 1 | None | Nausea | 2 | 0 | 0 |
| 24 | 34 | Musculoskeletal, photosensitivity | 135 | 1 | HQ | Treatment ongoing | 2 | 0 | 0 |
| 25∗† | 44 | Musculoskeletal | 3 | 1 | None | Infections | 2 | 0 | 0 |
| 26∗† | 29 | Musculoskeletal, alopecia | 2 | 2 | MTX | Nausea | 5 | 3 | 3 |
| 27 | 40 | Musculoskeletal, oral/genital ulcers, acute cutaneous lupus | 10 | 2 | HQ | Lack of efficacy | 5 | 0 | 0 |
∗Early cessation (≤3 months). †Deceased at the end of study period. HQ, hydroxychloroquine; MMF, mycophenolate mofetil; MTX, methotrexate.