Patient characteristics
| Patient no. . | Sex . | Age, y . | Rps19 . | History of response to steroids . | Ferritin level at inclusion, ng/mL* . | Arm . | Response . |
|---|---|---|---|---|---|---|---|
| 1 | F | 24 | Mutated | R | 1201 | B | N |
| 2 | M | 3 | Mutated | R | 1368 | A | N |
| 3 | F | 7 | Mutated | R | 837 | A | N |
| 4 | F | 3 | Wt | SHT | 767 | B | PR |
| 5 | F | 26 | Wt | SHT | 861 | A | N |
| 6 | M | 23 | Mutated | R | 8060 | B | N |
| 7 | M | 9 | Wt | S | 503 | A | N |
| 8 | F | 15 | Mutated | R | 1857 | B | N |
| 9 | F | 8 | Wt | R | 1061 | B | N |
| 10 | M | 3 | Mutated | R | 1229 | A | N |
| 11 | F | 8 | Wt | S | 1347 | A | N |
| 12 | F | 6 | Wt | S | 2568 | B | N |
| 13 | F | 12 | Mutated | R | 2014 | A | N |
| 14 | M | 4 | Wt | R | 1599 | A | N |
| 15 | F | 43 | Mutated | S | 736 | B | N |
| 16 | M | 12 | Wt | R | 2963 | B | N |
| 17 | M | 4 | Wt | SHT | 2032 | A | N |
| 18 | M | 13 | Mutated | SHT | 5319 | B | N |
| 19 | M | 4 | Wt | R | 714 | A | N |
| 20 | M | 7 | Mutated | SHT | 1125 | B | N |
| 21 | F | 25 | Mutated | S | 4000 | A | N |
| 22 | M | 16 | Mutated | S | 592 | A | N |
| 23 | M | 35 | Wt | R | 2569 | B | N |
| 24 | F | 19 | Wt | R | 1381 | B | N |
| 25 | F | 12 | Wt | R | 1063 | B | N |
| 26 | F | 14 | Wt | R | 567 | A | N |
| 27 | M | 21 | Wt | R | 793 | A | N |
| 28 | F | 36 | Wt | S | 1990 | B | PR |
| 29 | F | 8 | Mutated | SHT | 687 | A | N |
| 30 | M | 3 | Wt | R | 2131 | A | N |
| 31 | F | 38 | Mutated | R | 2290 | B | N |
| 32 | F | 20 | Mutated | R | 936 | B | N |
| 33 | M | 2 | Wt | R | 1670 | A | N |
| Patient no. . | Sex . | Age, y . | Rps19 . | History of response to steroids . | Ferritin level at inclusion, ng/mL* . | Arm . | Response . |
|---|---|---|---|---|---|---|---|
| 1 | F | 24 | Mutated | R | 1201 | B | N |
| 2 | M | 3 | Mutated | R | 1368 | A | N |
| 3 | F | 7 | Mutated | R | 837 | A | N |
| 4 | F | 3 | Wt | SHT | 767 | B | PR |
| 5 | F | 26 | Wt | SHT | 861 | A | N |
| 6 | M | 23 | Mutated | R | 8060 | B | N |
| 7 | M | 9 | Wt | S | 503 | A | N |
| 8 | F | 15 | Mutated | R | 1857 | B | N |
| 9 | F | 8 | Wt | R | 1061 | B | N |
| 10 | M | 3 | Mutated | R | 1229 | A | N |
| 11 | F | 8 | Wt | S | 1347 | A | N |
| 12 | F | 6 | Wt | S | 2568 | B | N |
| 13 | F | 12 | Mutated | R | 2014 | A | N |
| 14 | M | 4 | Wt | R | 1599 | A | N |
| 15 | F | 43 | Mutated | S | 736 | B | N |
| 16 | M | 12 | Wt | R | 2963 | B | N |
| 17 | M | 4 | Wt | SHT | 2032 | A | N |
| 18 | M | 13 | Mutated | SHT | 5319 | B | N |
| 19 | M | 4 | Wt | R | 714 | A | N |
| 20 | M | 7 | Mutated | SHT | 1125 | B | N |
| 21 | F | 25 | Mutated | S | 4000 | A | N |
| 22 | M | 16 | Mutated | S | 592 | A | N |
| 23 | M | 35 | Wt | R | 2569 | B | N |
| 24 | F | 19 | Wt | R | 1381 | B | N |
| 25 | F | 12 | Wt | R | 1063 | B | N |
| 26 | F | 14 | Wt | R | 567 | A | N |
| 27 | M | 21 | Wt | R | 793 | A | N |
| 28 | F | 36 | Wt | S | 1990 | B | PR |
| 29 | F | 8 | Mutated | SHT | 687 | A | N |
| 30 | M | 3 | Wt | R | 2131 | A | N |
| 31 | F | 38 | Mutated | R | 2290 | B | N |
| 32 | F | 20 | Mutated | R | 936 | B | N |
| 33 | M | 2 | Wt | R | 1670 | A | N |
F indicates female; M, male; Wt, wild type; R, strictly resistant to steroids; SHT, patient responder to steroids with a high threshold (>0.5 mg/kg/d); S, patient good responder to steroids in whom steroids were discontinued either because of side effects or loss of sensitivity; N, no response; PR, partial response.