Individual patients’ therapy and outcomes (N = 27)
| UPN . | No. of cladribine + Ara-C courses . | HS status and DAS at D1 cladribine + Ara-C . | Response and DAS after 2 courses . | Toxicity . | Time to NAD, d . | TT duration, y . | Cladribine cumulative dose, mg/m2 . | Reactivation /organ at reactivation . | Therapy of reactivation . | Duration of follow-up, y . | Vital status (cause of death) . | Sequelae . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1506648 | 2 | ADW 12 | ADB 8 | Hem: WHO 4 | 136 | 1.8 | 120 | None | 9.2 | L | ND MRI exophthalmia | |
| 1506863 | 2 | ADW 20 | ADB 15 | Hem: WHO 4 | MD | 0.2 | 90 | None | 9.2 | L | ||
| 1506802 | 2 | ADW 7 | ADB 2 | Hem: WHO 4 | 78 | 1.4 | 120 | None | 8.2 | L | DI | |
| 1506961 | 3 | ADW 13 | ADB 9 | Hem: WHO 4; PNP Aspergillus fumigatus | 181 | 2.0 | 215 | None | 7.9 | L | Development delay; not LCH related | |
| 1506962 | 2 | ADW 15 | ADB 8 | Hem: WHO 4 | 144 | 1.4 | 135 | None | 7.4 | L | ||
| 1507009 | 2 | ADW 8 | ADB 2 | Hem: WHO 4, skin rash, myalgia | 1.7 | 120 | None | 6.6 | L | |||
| 1506970 | 3 | ADW 9 | ADB 3 | Hem: WHO 4 long-term: condyloma | 128 | 3.3 | 195 | Skin | VBL + steroid for 18 mo | 6.8 | L | |
| 1507092 | 3 | ADW 19 | ADB 13 | Hem: WHO 4 gut: WHO 3 septicemia candida ICU | NA | NA | 165 | Liver, lung | Cladribine | 0.8 | D Reactivation | Sclerosing cholangitis |
| 1507202 | 3 | ADW 10 | ADB 2 | Hem: WHO 4 | NA | 1.7 | 380 | Liver, skin, spleen | Cladribine Ara-C HSCT | 1.7 | D (PNP D12 HSCT) | |
| 1507020 | 2 | ADW 15 | ADB 14 | Hem: WHO 4 gut: WHO 3 | NA | 2.8 | 198 | Liver, skin, spleen | Cladribine + Ara-C HSCT then VBL + steroid | 5.1 | L | |
| 1507220 | 2 | ADW 12 | ADS 4 | Hem: WHO 4 | MD | 1.6 | 210 | None | 3.2 | L | Mandible malformation | |
| 1507379 | 2 | ADW 8 | ADB 2 | Hem: WHO 4 sepsis Staphylococcus aureus | 59 | 1.9 | 120 | None | 4.6 | L | ||
| 1507361 | 2 | ADW 6 | NAD 0 | Hem: WHO 4 gut: WHO 3 septicemia, Clostridium difficile aspergillosis, ICU | 76 | 1.9 | 135 | None | 3.1 | L | ||
| 1507236 | 3 | ADS 12 | ADB 3 | Hem: WHO 4 tubulopathy HT, mucosis (G3) | 84 | 2.0 | 215 | None | 4.9 | L | ||
| 1507241 | 3 | ADW 16 | ADB 4 | Hem: WHO 4 ICU | MD | MD | 215 | None | 4.4 | L | Thyroid insufficiency | |
| 1507473 | 3 | ADW 9 | ADB 4 | Hem: WHO 4 | 144 | 1.9 | 165 | None | 4.1 | L | Sclerosing cholangitis OLT 3.8 y | |
| 1507255 | 2 | ADW 10 | ADB 3 | Hem: WHO 4 ICU | 64 | 3.8 | 315 | Liver, skin, spleen | Cladribine, Ara-C | 4.9 | L | |
| 3000050 | 3 | ADW 11 | ADB 7 | Hem: WHO 4 gut: WHO 3 | 172 | 2.0 | 225 | None | 5.9 | L | No | |
| 3000051 | 3 | ADW 13 | ADB 7 | Hem: WHO 4 | 82 | 4.9 | 225 | None | 5.4 | L | ||
| 3000052 | 3 | ADW 14 | ADB 3 | Hem: WHO 4 | 122 | 2.1 | 190 | None | 5.5 | L | ||
| 3000053 | 3 | ADW 6 | ADB 3 | Hem: WHO 4 gut: WHO 3 | NA | NA | 135 | None | 0.2 | D VZV D21 course 3 | ||
| 3000054 | 5 | ADW 19 | ADS 18 | Hem: WHO 4 | NA | NA | 245 | None | 0.7 | D pancytopenia Viral infection | ||
| 3000055 | 3 | ADW 6 | ADB 2 | Hem: WHO 4 | 364 | 2.0 | 185 | None | 3.4 | L | ND MRI | |
| 3000057 | 5 | ADW 14 | ADB 16 | Hem: WHO 4 gut: WHO 3 septicemia, ICU | 1971 | 5.4 | 269 | Skin | Skin 6MP MTX per os | 6.9 | L | |
| 3000058 | 4 | ADW 16 | NAD 0 | Hem: WHO 4 | 65 | 1.9 | 235 | None | 6,7 | L | ||
| 3000059 | 4 | ADW 9 | ADB 2 | Hem: WHO 4 pulmonary cavities (Aspergillus ?) | MD | 2.0 | 220 | None | 3,5 | L | Short stature no GHD | |
| 3000069 | 4 | ADW 14 | ADB 3 | Hem: WHO 4 | MD | 1.9 | 210 | None | 5,1 | L | Unilateral deafness, short stature (−2.5 SD)l | |
| UPN . | No. of cladribine + Ara-C courses . | HS status and DAS at D1 cladribine + Ara-C . | Response and DAS after 2 courses . | Toxicity . | Time to NAD, d . | TT duration, y . | Cladribine cumulative dose, mg/m2 . | Reactivation /organ at reactivation . | Therapy of reactivation . | Duration of follow-up, y . | Vital status (cause of death) . | Sequelae . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1506648 | 2 | ADW 12 | ADB 8 | Hem: WHO 4 | 136 | 1.8 | 120 | None | 9.2 | L | ND MRI exophthalmia | |
| 1506863 | 2 | ADW 20 | ADB 15 | Hem: WHO 4 | MD | 0.2 | 90 | None | 9.2 | L | ||
| 1506802 | 2 | ADW 7 | ADB 2 | Hem: WHO 4 | 78 | 1.4 | 120 | None | 8.2 | L | DI | |
| 1506961 | 3 | ADW 13 | ADB 9 | Hem: WHO 4; PNP Aspergillus fumigatus | 181 | 2.0 | 215 | None | 7.9 | L | Development delay; not LCH related | |
| 1506962 | 2 | ADW 15 | ADB 8 | Hem: WHO 4 | 144 | 1.4 | 135 | None | 7.4 | L | ||
| 1507009 | 2 | ADW 8 | ADB 2 | Hem: WHO 4, skin rash, myalgia | 1.7 | 120 | None | 6.6 | L | |||
| 1506970 | 3 | ADW 9 | ADB 3 | Hem: WHO 4 long-term: condyloma | 128 | 3.3 | 195 | Skin | VBL + steroid for 18 mo | 6.8 | L | |
| 1507092 | 3 | ADW 19 | ADB 13 | Hem: WHO 4 gut: WHO 3 septicemia candida ICU | NA | NA | 165 | Liver, lung | Cladribine | 0.8 | D Reactivation | Sclerosing cholangitis |
| 1507202 | 3 | ADW 10 | ADB 2 | Hem: WHO 4 | NA | 1.7 | 380 | Liver, skin, spleen | Cladribine Ara-C HSCT | 1.7 | D (PNP D12 HSCT) | |
| 1507020 | 2 | ADW 15 | ADB 14 | Hem: WHO 4 gut: WHO 3 | NA | 2.8 | 198 | Liver, skin, spleen | Cladribine + Ara-C HSCT then VBL + steroid | 5.1 | L | |
| 1507220 | 2 | ADW 12 | ADS 4 | Hem: WHO 4 | MD | 1.6 | 210 | None | 3.2 | L | Mandible malformation | |
| 1507379 | 2 | ADW 8 | ADB 2 | Hem: WHO 4 sepsis Staphylococcus aureus | 59 | 1.9 | 120 | None | 4.6 | L | ||
| 1507361 | 2 | ADW 6 | NAD 0 | Hem: WHO 4 gut: WHO 3 septicemia, Clostridium difficile aspergillosis, ICU | 76 | 1.9 | 135 | None | 3.1 | L | ||
| 1507236 | 3 | ADS 12 | ADB 3 | Hem: WHO 4 tubulopathy HT, mucosis (G3) | 84 | 2.0 | 215 | None | 4.9 | L | ||
| 1507241 | 3 | ADW 16 | ADB 4 | Hem: WHO 4 ICU | MD | MD | 215 | None | 4.4 | L | Thyroid insufficiency | |
| 1507473 | 3 | ADW 9 | ADB 4 | Hem: WHO 4 | 144 | 1.9 | 165 | None | 4.1 | L | Sclerosing cholangitis OLT 3.8 y | |
| 1507255 | 2 | ADW 10 | ADB 3 | Hem: WHO 4 ICU | 64 | 3.8 | 315 | Liver, skin, spleen | Cladribine, Ara-C | 4.9 | L | |
| 3000050 | 3 | ADW 11 | ADB 7 | Hem: WHO 4 gut: WHO 3 | 172 | 2.0 | 225 | None | 5.9 | L | No | |
| 3000051 | 3 | ADW 13 | ADB 7 | Hem: WHO 4 | 82 | 4.9 | 225 | None | 5.4 | L | ||
| 3000052 | 3 | ADW 14 | ADB 3 | Hem: WHO 4 | 122 | 2.1 | 190 | None | 5.5 | L | ||
| 3000053 | 3 | ADW 6 | ADB 3 | Hem: WHO 4 gut: WHO 3 | NA | NA | 135 | None | 0.2 | D VZV D21 course 3 | ||
| 3000054 | 5 | ADW 19 | ADS 18 | Hem: WHO 4 | NA | NA | 245 | None | 0.7 | D pancytopenia Viral infection | ||
| 3000055 | 3 | ADW 6 | ADB 2 | Hem: WHO 4 | 364 | 2.0 | 185 | None | 3.4 | L | ND MRI | |
| 3000057 | 5 | ADW 14 | ADB 16 | Hem: WHO 4 gut: WHO 3 septicemia, ICU | 1971 | 5.4 | 269 | Skin | Skin 6MP MTX per os | 6.9 | L | |
| 3000058 | 4 | ADW 16 | NAD 0 | Hem: WHO 4 | 65 | 1.9 | 235 | None | 6,7 | L | ||
| 3000059 | 4 | ADW 9 | ADB 2 | Hem: WHO 4 pulmonary cavities (Aspergillus ?) | MD | 2.0 | 220 | None | 3,5 | L | Short stature no GHD | |
| 3000069 | 4 | ADW 14 | ADB 3 | Hem: WHO 4 | MD | 1.9 | 210 | None | 5,1 | L | Unilateral deafness, short stature (−2.5 SD)l | |
ADB, active disease, better; ADS, active disease, stable; ADW, active disease, worse; D, dead; DAS, disease activity score10 ; DI, diabetes insipidus; GHD, growth hormone deficiency; Hem, hematological; HS, hematopoeitic stem cell transplantation; HT, hypertension; ICU, intensive care unit; L, living; MD, missing data; NA, not achieved; NAD, nonactive disease; ND MRI, neuro degenerative MRI features; OLT, orthotopic liver transplantation; PNP, pneumonitis; UPN, unique patient number; VZV, varicella zoster virus; WHO, World Health Organization (scale for side effects).