Patient characteristics
| Patient no. . | Year of HSCT/Center . | Sex . | Onset type . | Age at onset . | Age at HSCT . | Treatment pre-HSCT . | Comorbidities associated with JIA treatment . |
|---|---|---|---|---|---|---|---|
| 1 | 2007/GOSH | F | Poly- JIA (RF−) | 24 mo | 10 y 1 mo | ASCT, MTX, CsA, etanercept, tocilizumab, anakinra, thalidomide, FK506 | Growth failure-on growth hormone |
| 2 | 2009/CCHMC | F | sJIA + MAS | 36 mo | 4 y 6 mo | Corticosteroids, CsA | Obesity due to corticosteroids excess, MAS |
| 3 | 2009/GOSH | F | sJIA + MAS | 5 mo | 2 y 8 mo | MTX, corticosteroids, anakinra, CsA, infliximab | CMV viremia |
| 4 | 2010/GOSH | F | sJIA + MAS | 5 y 7 mo | 7 y 8 mo | Etoposide, CsA, corticosteroids, anakinra, ATG | Adenoviremia, poor left ventricular function with EF of 20%+ small pericardial effusion. Had 2 PICU admissions: 1. cardiac failure at presentation of MAS and 2. Pseudomonas sepsis |
| 5 | 2012/N | F | Poly-JIA (RF−) | 10 mo | 13 y 3 mo | Corticosteroids, MTX, MMF, etanercept, infliximab, adalimumab, abatacept, anakinra, rituximab | Severe corticosteroids-related toxicity (osteoporosis with multiple vertebral crush fractures, cushingoid features, right eye glaucoma and cataract surgery), poor QOL |
| 6 | 2014/N | M | Poly-JIA (RF−) | 19 mo | 3 y 3 mo | Corticosteroids, MTX, infliximab, immunoglobulin replacement | Undefined primary immunodeficiency-early childhood interstitial pneumonitis (biopsy proven, PICU, home O2), colitis, hypogammaglobulinemia, skin rashes, nail dystrophy, fevers (no underlying mutation found so far) |
| 7 | 2014/N | F | sJIA | 24 mo | 6.5 y | Corticosteroids, MTX, tocilizumab, infliximab, MTX, anakinra | Corticosteroids-related growth failure, cushingoid features, poor QOL |
| 8 | 2014/UCLH | M | Poly-JIA (RF−) | 18 mo | 16 y 9 mo | Corticosteroids, MTX, adalumimab, abatecept, tocilizumab | Joint destruction due to refractory disease |
| 9 | 2014/GOSH | M | sJIA + MAS | 14 mo | 6 y 2 mo | MTX, high-dose corticosteroids, anakinra, tocilizumab | Chest infection + pleural effusion requiring mechanical ventilation. |
| Seizures secondary to steroid induced hypertensive encephalopathy, MAS | |||||||
| 10 | 2014/UCLH | M | sJIA | 18 mo | 16 y 9 mo | Infliximab (allergic), tocilizumab, canakinumab, MTX | MAS, renal impairment due to ciclosporin, osteoporosis, cataracts, renal stones, warts, growth and pubertal delay (but coexistent Kinefelter syndrome) |
| 11 | 2014/GOSH | F | sJIA + MAS | 12 y | 13 y 3 mo | Corticosteroids, CsA, anakinra | Mild left ventricular dysfunction. FS: 23%, MAS |
| 12 | 2014/Prague | F | Poly-JIA (RF−) | 4 y | 9 y 2 mo | Corticosteroids, etanercept, tocilizumab, adalimumab, anakinra, abatacept, rituximab, canakinumab | Cushingoid features due to corticosteroids excess, hypertension, severe osteoporosis with vertebral crush fractures, growth failure |
| 13 | 2014/CCHMC | F | sJIA | 9 mo | 4 y 4 mo | Corticosteroids, anakinra, rituximab, tocilizumab, CsA | Overweight due to corticosteroids excess |
| 14 | 2015/CCHMC | F | sJIA | 12 y | 16 y 9 mo | Corticosteroids, adalimumab, tociluzumab, canakinumab, rituximab, MTX, abatacept | Osteoporosis, obesity, glucose intolerance, polycystic ovarian syndrome, nonalcoholic hepatic steatosis |
| 15 | 2016/N | F | sJIA | 18 mo | 11 y | Corticosteroids, MTX, etanercept, anakinra, adalimumab, tocilizumab, infliximab | Corticosteroids-related osteoporosis (impact-fracture) and growth failure, elevated serum amyloid-A |
| 16 | 2016/N | F | sJIA | 2 y | 5 y | Corticosteroids, MTX, etanercept, adalimumab, tocilizumab, infliximab, anakinra | Poor QOL |
| Patient no. . | Year of HSCT/Center . | Sex . | Onset type . | Age at onset . | Age at HSCT . | Treatment pre-HSCT . | Comorbidities associated with JIA treatment . |
|---|---|---|---|---|---|---|---|
| 1 | 2007/GOSH | F | Poly- JIA (RF−) | 24 mo | 10 y 1 mo | ASCT, MTX, CsA, etanercept, tocilizumab, anakinra, thalidomide, FK506 | Growth failure-on growth hormone |
| 2 | 2009/CCHMC | F | sJIA + MAS | 36 mo | 4 y 6 mo | Corticosteroids, CsA | Obesity due to corticosteroids excess, MAS |
| 3 | 2009/GOSH | F | sJIA + MAS | 5 mo | 2 y 8 mo | MTX, corticosteroids, anakinra, CsA, infliximab | CMV viremia |
| 4 | 2010/GOSH | F | sJIA + MAS | 5 y 7 mo | 7 y 8 mo | Etoposide, CsA, corticosteroids, anakinra, ATG | Adenoviremia, poor left ventricular function with EF of 20%+ small pericardial effusion. Had 2 PICU admissions: 1. cardiac failure at presentation of MAS and 2. Pseudomonas sepsis |
| 5 | 2012/N | F | Poly-JIA (RF−) | 10 mo | 13 y 3 mo | Corticosteroids, MTX, MMF, etanercept, infliximab, adalimumab, abatacept, anakinra, rituximab | Severe corticosteroids-related toxicity (osteoporosis with multiple vertebral crush fractures, cushingoid features, right eye glaucoma and cataract surgery), poor QOL |
| 6 | 2014/N | M | Poly-JIA (RF−) | 19 mo | 3 y 3 mo | Corticosteroids, MTX, infliximab, immunoglobulin replacement | Undefined primary immunodeficiency-early childhood interstitial pneumonitis (biopsy proven, PICU, home O2), colitis, hypogammaglobulinemia, skin rashes, nail dystrophy, fevers (no underlying mutation found so far) |
| 7 | 2014/N | F | sJIA | 24 mo | 6.5 y | Corticosteroids, MTX, tocilizumab, infliximab, MTX, anakinra | Corticosteroids-related growth failure, cushingoid features, poor QOL |
| 8 | 2014/UCLH | M | Poly-JIA (RF−) | 18 mo | 16 y 9 mo | Corticosteroids, MTX, adalumimab, abatecept, tocilizumab | Joint destruction due to refractory disease |
| 9 | 2014/GOSH | M | sJIA + MAS | 14 mo | 6 y 2 mo | MTX, high-dose corticosteroids, anakinra, tocilizumab | Chest infection + pleural effusion requiring mechanical ventilation. |
| Seizures secondary to steroid induced hypertensive encephalopathy, MAS | |||||||
| 10 | 2014/UCLH | M | sJIA | 18 mo | 16 y 9 mo | Infliximab (allergic), tocilizumab, canakinumab, MTX | MAS, renal impairment due to ciclosporin, osteoporosis, cataracts, renal stones, warts, growth and pubertal delay (but coexistent Kinefelter syndrome) |
| 11 | 2014/GOSH | F | sJIA + MAS | 12 y | 13 y 3 mo | Corticosteroids, CsA, anakinra | Mild left ventricular dysfunction. FS: 23%, MAS |
| 12 | 2014/Prague | F | Poly-JIA (RF−) | 4 y | 9 y 2 mo | Corticosteroids, etanercept, tocilizumab, adalimumab, anakinra, abatacept, rituximab, canakinumab | Cushingoid features due to corticosteroids excess, hypertension, severe osteoporosis with vertebral crush fractures, growth failure |
| 13 | 2014/CCHMC | F | sJIA | 9 mo | 4 y 4 mo | Corticosteroids, anakinra, rituximab, tocilizumab, CsA | Overweight due to corticosteroids excess |
| 14 | 2015/CCHMC | F | sJIA | 12 y | 16 y 9 mo | Corticosteroids, adalimumab, tociluzumab, canakinumab, rituximab, MTX, abatacept | Osteoporosis, obesity, glucose intolerance, polycystic ovarian syndrome, nonalcoholic hepatic steatosis |
| 15 | 2016/N | F | sJIA | 18 mo | 11 y | Corticosteroids, MTX, etanercept, anakinra, adalimumab, tocilizumab, infliximab | Corticosteroids-related osteoporosis (impact-fracture) and growth failure, elevated serum amyloid-A |
| 16 | 2016/N | F | sJIA | 2 y | 5 y | Corticosteroids, MTX, etanercept, adalimumab, tocilizumab, infliximab, anakinra | Poor QOL |
ATG, antithymocyte globulin; CCHMC, Cincinnati Children’s Hospital Medical Center; CMV, cytomegalovirus; EF, ejection fraction; F, female; FS, fraction of shortening; GOSH, Great Ormond Street Hospital; M, male; N, Newcastle Great North Children’s Hospital; PICU, pediatric intensive care unit; QOL, quality of life; UCLH, University College London Hospital.