Table 1 Clinical data documentation (n = 33, 35 samples). Demographics and clinical characteristics of patients, obtained from clinical health records submitted to digital hospital information systems in the participating centers in Berlin, Erlangen, Essen, Göttingen, Paris and Zurich. Patient ID from which multiple biopsies have been sampled are indicated with a second numbering system (e.g. “19.1”). For improved readability, patient characteristics have been abbreviated: NDC (A), Mi2 subtype (B), TIF1γ subtype (C), and irMyositis (D) groups; monotherapy with Pembrolizumab (Pembro), Nivolumab (Nivo) or Ipilimumab (Ipi), as well as a combination approach of Ipilimumab/Nivolumab (Com.); muscle pathology characteristics typically found in dermatomyositis (DMS.) or necrotizing myopathy (N.M.); patient information not reported by the center or data is not part of routine patient assessment (N.R.); not-applicable or unperformed treatment or diagnostic approach (N/A); no treatment (steroids or immunosuppressants) at time of biopsy (N.T.); no autoantibody found (Neg.); age at time of biopsy (Age); body weight (b.w.); methylprednisolone (mp.); ophthalmoplegia (op.); tetraparesis (tp.); hemiplegia (hp.); axial weakness (aw.); cutaneous Melanoma stage IV (C.M. IV) and Cutaneous melanoma stage not reported (C.M. S.N.R.); endometrioid adenocarcinoma (E.A.); skin lesions (S.L.); bulbar involvement (B.I.). Further commentaries are abbreviated by #: steroids, IVIG, plasmapheresis, hemodialysis, intensive care treatment; ##: left hip flexor palsy M4 and hyposensitivity ventral thigh; ###: MGUS/metastatic melanoma (subcutaneous and soft tissue metastases).
Group | Patient ID | Muscle Pathology Characteristics | Autoantibody Profiles | Type of ICI Therapy | Treatment at Time of Biopsy | Sex (M/F) | Age | Muscle Strength Assessments | Ck Level (U/I) | Comorbidities/Malignancy | Extramuscular Manifestations |
|---|---|---|---|---|---|---|---|---|---|---|---|
A | 1 | normal | N.R. | N/A | N.R. | F | 39 | N.R. | normal | N.R. | N.R. |
2 | normal | N.R. | N/A | N.R. | M | 64 | N.R. | normal | N.R. | N.R. | |
3 | normal | Neg. | N/A | N.T. | M | 22 | normal | 127 | none | none | |
4 | normal | Neg. | N/A | N.T. | F | 49 | normal | normal | none | none | |
B | 5 | DMS. | anti-Mi-2 | N/A | N.R. | F | 86 | proximal tp. | N.R. | N.R. | S.L. |
6 | DMS. | anti-Mi-2 | N/A | N.R. | F | 83 | proximal tp. | 9,000 | N.R. | S.L. | |
7 | DMS. | anti-Mi-2 | N/A | N.T. | M | 54 | 4/5 diplegia (arms) | N.R. | none | S.L. | |
8 | DMS. | anti-Mi-2 | N/A | N.R. | F | 82 | 4/5 tp. | N.R. | none | S.L. | |
9 | DMS. | anti-Mi-2 | N/A | N.R. | F | 62 | 4/5 tp. | 6,400 | none | S.L. | |
10 | DMS. | anti-Mi-2 | N/A | N.T. | F | 48 | N.R. | N.R. | none | S.L. | |
11 | DMS. | anti-Mi-2 | N/A | N.T. | M | 57 | 4/5 proximal hp | N.R. | none | S.L. | |
12 | DMS. | anti-Mi-2 | N/A | N.T. | F | 86 | 4/5 proximal hp | N.R. | N.R. | S.L. | |
C | 13 | DMS. | anti-TIF-1γ | N/A | N.T. | F | 62 | 3–4/5 tp. | N.R. | breast cancer | S.L., B.I. |
14 | DMS. | anti-TIF-1γ | N/A | N.T. | M | 71 | 4/5 tp. | 2,000 | none | S.L. | |
15 | DMS. | anti-TIF-1γ | N/A | N.T. | F | 60 | 4/5 tp. | 2,400 | E.A. | S.L. | |
16 | DMS. | anti-TIF-1γ | N/A | N.T. | F | 48 | 4/5 tp. | N.R. | N.R. | none | |
17 | DMS. | anti-TIF-1γ | N/A | N.T. | F | 69 | 4/5 tp. | 11,500 | N.R. | none | |
18 | DMS | anti-TIF-1γ | N/A | N.T. | F | 64 | N.R. | N.R. | none | skin calcinosis | |
D | 19.1 | N.M. | Neg. | Com. | N.R. | M | 75 | proximal tp. | N.R. | C.M. S.N.R. | N.R. |
19.2 | N.M. | Neg. | Com. | N.R. | M | 75 | proximal tp. | N.R. | C.M. S.N.R. | N.R. | |
20 | signs of inflammation | Neg. | Pembro | steroids | M | 75 | multiple## | 6,870 | multiple### | myocarditis | |
21 | N.R. | Neg. | Pembro | prednisolone 0.5 mg/kg b.w | M | 75 | N/A | 1,626 | C.M. IV | transaminitis, myocarditis | |
22 | N.R. | N/A | Com. | mp. 1 mg/kg b.w | M | 69 | N/A | 301 | C.M. IV | hepatitis G3, thyreoiditis | |
23.1 | lymphocytic infiltrates | anti-cN-1A | Ipi, Pembro | steroids | M | 58 | proximal hp. | 2,400 | C.M. IV | none | |
23.2 | lymphocytic infiltrates | anti-cN-1A | Ipi, Pembro | steroids | M | 58 | proximal hp. | 2,400 | C.M. IV | none | |
24 | N.M. | Neg. | Pembro | mp. 1000 mg | M | 61 | 5/5 peripheral extremities, aw., op. | 11,900 | C.M. S.N.R. | vitiligo | |
25 | N.M. | Neg. | Nivo | mp. 500 mg | M | 74 | upper legs and aw., diplopia | 6,200 | lung cancer | B.I. | |
26 | N.M. | Neg. | Nivo/anti-TIGIT | N.T. | F | 63 | 4/5 hp. | 2,705 | renal cancer | B.I., dyspnea | |
27 | N.M. | Neg. | Pembro | N.T. | F | 86 | aw., op., ptosis | 1,400 | C.M. S.N.R. | ptosis | |
28 | N.M. | Neg. | Pembro, Nivo | N.T. | M | 75 | aw. | N.R. | C.M. S.N.R. | none | |
29 | N.M. | Neg. | Nivo | N.T. | F | 76 | tp. | N.R. | C.M. S.N.R. | none | |
30 | N.M. | Neg. | Nivo | N.T. | M | 83 | upper legs and aw. | 1,059 | C.M. S.N.R. | none | |
31 | signs of atrophy and inflammation | Neg. | Com. | steroids | F | 59 | N.R | 625 | C.M. IV | colitis, arthritis | |
32 | N.M. | Neg. | Com. | multiple# | M | 77 | N.R | > 42,670 | C.M. S.N.R. | none | |
33 | N.M. | Neg. | Nivo | N.R. | M | 84 | tp. | N.R. | C.M. S.N.R. | N.R. |
