Table 3.
Patients with monogenic disease who responded to immunosuppression
| Patient | Gene | Sex | Age at onset, yr | Resistance to Steroids | First Biopsy | CKD Stage | Extrarenal Phenotype | Length of Follow-Up, yr | Medication for which There Was Response | Response |
|---|---|---|---|---|---|---|---|---|---|---|
| 7656 | WT1 | M | 3 | Primary | Diffuse mesangial sclerosis | 2 | Denys–Drash syndrome | 1.6 | MMF, tacrolimus | Complete, complete |
| 495 | NPHS1 | F | 2 | Primary | Minimal change disease | 2 | No | 17.8 | MMF | Partial |
| 514 | SMARCAL1 | M | 7 | Primary | FSGS | 1 | No | 0.4 | Cyclosporin | Partial |
| 687 | CRB2 | F | 0 | Presumed | Minimal change disease | 1 | No | 4.0 | Levamisole | Partial |
| 729 | NPHS2 | M | 7 | Primary | Other | 2 | Asthma | 2.8 | Tacrolimus | Partial |
| 731 | MAGI2 | M | 0 | Primary | Minimal change disease | 1 | Pyloric stenosis, polydactyly, thrombocytosis | 11.7 | Cyclosporin | Partial |
| 770 | COL4A3 | F | 7 | Primary | FSGS | 1 | No | 2.5 | Cyclosporin | Partial |
| 811 | WT1 | F | 3 | Primary | Alport syndrome | 1 | Chronic cough and diarrhea, Frasier syndrome | 1.1 | Tacrolimus | Partial |
| 900 | LMX1B | F | 14 | Primary | FSGS | 1 | Delayed puberty | 1.1 | Tacrolimus | Partial |
M, male; MMF, mycophenolate mofetil; F, female. For correlation of UK National Registry of Rare Kidney Diseases numbers with previously published identification numbers, see Supplemental Table 3.