Table 2.
| Study | Year | N° pts | Purpose | Population | Histology/PR expression | Intervention | Outcome and results | Side effects | Limitations | Conclusion |
|---|---|---|---|---|---|---|---|---|---|---|
| Grunberg et al. [29] | 1991 | 14 | Efficacy of long-term oral therapy with mifepristone in unresectable meningiomas 11 pts were progressing (radiologically or symptomatically) before starting the TT with mifepristone |
Inoperable meningiomas 6 M 8 F: (i) 2 premenop (ii) 6 postmenop |
5 meningothelial 3 cellular 2 fibrous meningioma 2 malignant meningioma 2 w/o biopsy No evaluation of PR expression |
Mifepristone 200 mg/d for 2–31 mo (9 pts more than 12 mo) Dexa 1 mg daily during the first 14 d of treatment |
13 pts assessed for response (1 refused) (i) 5/13 pts radiological regression after at least 6 mo (ii) 3/13 subjective clinical improvement after 2-3 mo (iii) 3/13 progression (of which 2 were malignant meningiomas) |
14 evaluated (i) 11/14 fatigue (ii) 5/14 hot flashes (iii) 3/6 M gynecomastia (iv) 2 alopecia (v) 2 amenorrhea (premenop F) (vi) 1 hypothyroidism |
Subjective clinical evaluation Radiological response evaluated with CT or MRI Heterogenous population: (i) 2 premenop F (ii) 6 postmenop F (iii) 6 men No evaluation of PR expression 1 previous RT 4 previous hormonal therapy (MPA and tamoxifen) For the 3 pts defined as stable before starting mifepristone, the previous FU is ns |
PRO |
|
| ||||||||||
| Grunberg et al. [30] | 2006 | 28 (Included the 14 pts of the study of Grunberg et al. 1991 [29]) | Clinical tolerance of long-term treatment with mifepristone | 9 M 19 F: (i) 5 premenop (ii) 14 postmenop |
13 meningothelial 4 fibroblastic 5 cellular/ns 2 malignant 4 not biopsied No evaluation of PR expression |
Mifepristone 200 mg/d for 2–31 mo Mean FU 35 mo (2–157 mo) Total of 1626 patient-months Dexa 1 mg daily during the first 14 d of treatment |
8/28 pts radiological (5) and clinical (3) improvement (7/8 were male or premenopausal F) |
22/28 pts mild fatigue 13/28 pts hot flashes 6/28 pts gynecomastia 3/28 depression 1 N/V 3/28 pts endometrial hyperplasia 1 pt peritoneal adenocarcinoma |
2 malignant meningiomas 4 no histological diagnosis No evaluation of PR expression |
CON |
|
| ||||||||||
| Lamberts et al. [31] | 1992 | 10 | Effect of mifepristone treatment in recurrent or inoperable meningiomas recent evidence of growing: neuroradiological in 9 cases ophthalmological in 4 cases |
12 recurrent or inoperable meningiomas in 10 pts 7 pts previous operation 3 males 1 premenop F (with NF2) 6 postmenop F |
No data about histology nor PR expression | Mifepristone 200 mg/d for 12 mo 4 cases started Dexa 7.5 mg daily and continued throughout the study |
5/12 meningiomas grew (2 deaths) 3/12 stable 4/12 transient regression (progression at discontinuation of TT) 5/10 pts symptomatic improvement objective assessment in 2 pts 1 pt continued to deteriorate |
Asthenia, N/V | CT scan evaluation (planimetry) Group of aggressive meningiomas (recent evidence of growing) No data about histology nor PR expression Graphic over responsive patients Analyzable in different ways |
PRO |
|
| ||||||||||
| De Keizer and Smit [32] | 2004 | 2 | Efficacy of long-term mifepristone treatment | Unresectable sphenoid-ridge meningioma 2 F: 1 postmenop F |
Both tumors were PR + | Mifepristone 200 mg/d then increased to 400 mg/d Dexa 0.5 mg three times daily FU 14 y |
2 controlled tumor growth 1 improved VA 1 improved headache, stable VA clinical deterioration, and radiological growth after TT discontinuation Initial benefits persist |
1 endometrial hyperplasia | CT and MRI evaluation | PRO |
|
| ||||||||||
| Touat et al. [33] | 2014 | 3 | Efficacy of mifepristone in meningiomatosis | F, postmenop | Histological examination in one case | Mifepristone 200 mg/d. FU 4–10 y |
3/3 long lasting clinical and 2/3 radiological response or stabilization | 1 benign ovarian serous cystadenoma | Small n° pts Histological examination just in one case |
PRO |
|
| ||||||||||
| Spitz et al. [34] | 2005 | 25 | To determine clinical side effects and biochemical abnormalities with long-term mifepristone therapy | 16 F and 9 M 22–80 y unresectable meningioma |
Mifepristone 200 mg/d (follow-up variable from 13 y to 4 mo) Total of 1620 mo |
Safety of long-term TT | (i) 22/25 pts fatigue (ii) 2 endometrial hyperplasia (iii) 2 endometrial thickening |
Varied follow-up Heterogeneous cohort: 5 premenopausal F, 11 postmenopausal F, and 9 M |
PRO | |
|
| ||||||||||
| Grunberg et al. [35] | 2001 | 160 (80 per arm) | Efficacy of mifepristone in unresectable meningiomas | Adults with unresectable histologically confirmed nonmalignant meningiomas appeared (22%) or progressing (78%) within 2 y Median age 57 y 30% M 19% premenop F 51% postmenop F 29% prior RTH |
ns | Blinded administration of mifepristone 200 mg/d versus placebo Follow-up 2 y |
FFP (freedom from progression) Progression defined as anatomic growth or neurologic deterioration No significant difference in response between the arms: median FFP was 10 mo for mifepristone pts and 12 mo for placebo pts (p = 0.44) |
Fatigue (72% mifepristone pts vs 54% placebo pts) Headache Hot flashes 9 mifepristone pts had endometrial hyperplasia (16% F) |
Limited data available (abstract) No information about histological type and PR expression |
CON |
CON: against; d: days; dexa: dexamethasone; EGF: epithelial growth factor; ER: estrogen receptors; F: female; M: male; mo: months; N°: number; ns: not specified; N/V: nausea and vomiting; PR: progesterone receptors; Postmenop: postmenopausal; Premenop: premenopausal; PRO: for, in favor of; Pts: patients; TT: treatment; VA: visual acuity; y: years; w: weeks; w/o: without.