Patient characteristics
| Case . | Sex . | Age at diagnosis, y . | Age at IM initiation, y . | IM duration, mo . | IM maximum dose . | Previous treatment . | IM indication . | ECD involvement . | Outcome of IM . | IM failure . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | M | 36 | 40 | 12 | 200 mg/d | Steroids, Vinblastine, MTX, ASCT | Failure of other therapies | Diabetes insipidus; panhypopituitarism; xanthelasma; “coated aorta”; pericardium, left coronary artery; left hydronephrosis; retroorbital masses; lung fibrosis | Stability of all ECD involvement sites; did not tolerate higher doses; treatment stopped by patient | Stable |
| 2 | F | 63 | 63 | 15 | 600 mg/d | None | CNS and cardiovascular involvements; psychiatric contraindication to IFNα | Diabetes insipidus; severe ataxia (cerebellar mass); hypophysitis; exophthalmos; “coated aorta” | Absence of efficacy after 3 mo of treatment at 200 mg/d justifying adjunction of IFNα (3 M × 3) due to severity of the disease; brain MRI stable; IFNα not well tolerated (fever, psychiatric disorders) and stopped after 10 mo; worsening of cerebellar involvement, leading to treatment discontinuation. | Worsening |
| 3 | F | 62 | 63 | 7 | 800 mg/d | None | CNS and cardiovascular involvements | Severe CNS with several focal lesions (“pseudo-meningioma”), ataxia; “pseudo-atrial” tumor; xanthelasma; lung fibrosis; bone pain | Absence of occurrence of new brain focal lesion (the patient had undergone four operations on the brain in the 5 y before treatment initiation), initially better, but worsening of ataxia and bone pain during last 2 mo of treatment; TEP-FDG: new cerebral fixation uptake appeared under treatment, while brain MRI and echocardiography results were stable | Initial stabilization of the disease, before worsening |
| 4 | M | 60 | 62 | 24 | 200 mg/d | Steroids | Cardiovascular involvement | * Bone pain; “coated aorta,” celiac trunk, superior mesenteric artery, left subclavian artery, coronaropathy | Persistence of bone pain; history of myocardial infarction in May 2006 (coronary stent); did not tolerate higher doses (visual disturbance). | Worsening |
| 5 | M | 41 | 46 | 6 | 300 mg/d | Steroids, IFNα, MMF, MTX, ASCT | Failure of other therapies; CNS and cardiovascular involvements | Bone pain; periaortic fibrosis, renovascular HT; severe CNS, ataxia; Hypophysitis | Worsening of ataxia, brain MRI stable; Septic osteomyelitis of the right jaw leading to treatment discontinuation | Worsening |
| 6 | M | 18 | 31 | 15 | 400 mg/d | 2CDA, tandem ASCT | Recurrence 6 y after other therapies | Massive exophthalmos; Voluminous facial mass involving both orbits and the facial sinuses after a 6 y remission after ASCT | Absence of worsening of exophthalmos, which reappeared 6 y after ASCT and remains mild to moderate | Stable |
| Case . | Sex . | Age at diagnosis, y . | Age at IM initiation, y . | IM duration, mo . | IM maximum dose . | Previous treatment . | IM indication . | ECD involvement . | Outcome of IM . | IM failure . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | M | 36 | 40 | 12 | 200 mg/d | Steroids, Vinblastine, MTX, ASCT | Failure of other therapies | Diabetes insipidus; panhypopituitarism; xanthelasma; “coated aorta”; pericardium, left coronary artery; left hydronephrosis; retroorbital masses; lung fibrosis | Stability of all ECD involvement sites; did not tolerate higher doses; treatment stopped by patient | Stable |
| 2 | F | 63 | 63 | 15 | 600 mg/d | None | CNS and cardiovascular involvements; psychiatric contraindication to IFNα | Diabetes insipidus; severe ataxia (cerebellar mass); hypophysitis; exophthalmos; “coated aorta” | Absence of efficacy after 3 mo of treatment at 200 mg/d justifying adjunction of IFNα (3 M × 3) due to severity of the disease; brain MRI stable; IFNα not well tolerated (fever, psychiatric disorders) and stopped after 10 mo; worsening of cerebellar involvement, leading to treatment discontinuation. | Worsening |
| 3 | F | 62 | 63 | 7 | 800 mg/d | None | CNS and cardiovascular involvements | Severe CNS with several focal lesions (“pseudo-meningioma”), ataxia; “pseudo-atrial” tumor; xanthelasma; lung fibrosis; bone pain | Absence of occurrence of new brain focal lesion (the patient had undergone four operations on the brain in the 5 y before treatment initiation), initially better, but worsening of ataxia and bone pain during last 2 mo of treatment; TEP-FDG: new cerebral fixation uptake appeared under treatment, while brain MRI and echocardiography results were stable | Initial stabilization of the disease, before worsening |
| 4 | M | 60 | 62 | 24 | 200 mg/d | Steroids | Cardiovascular involvement | * Bone pain; “coated aorta,” celiac trunk, superior mesenteric artery, left subclavian artery, coronaropathy | Persistence of bone pain; history of myocardial infarction in May 2006 (coronary stent); did not tolerate higher doses (visual disturbance). | Worsening |
| 5 | M | 41 | 46 | 6 | 300 mg/d | Steroids, IFNα, MMF, MTX, ASCT | Failure of other therapies; CNS and cardiovascular involvements | Bone pain; periaortic fibrosis, renovascular HT; severe CNS, ataxia; Hypophysitis | Worsening of ataxia, brain MRI stable; Septic osteomyelitis of the right jaw leading to treatment discontinuation | Worsening |
| 6 | M | 18 | 31 | 15 | 400 mg/d | 2CDA, tandem ASCT | Recurrence 6 y after other therapies | Massive exophthalmos; Voluminous facial mass involving both orbits and the facial sinuses after a 6 y remission after ASCT | Absence of worsening of exophthalmos, which reappeared 6 y after ASCT and remains mild to moderate | Stable |