Salvage therapy for relapsed/refractory Langerhans cell histiocytosis
| Regimen | Study (n) | Response | SR | Remark |
|---|---|---|---|---|
| 2-CdA (5 mg/m2×5 d) [33] | Prospective | RO+ 22% (RR) | 2-yr OS | 2-CdA for RO-multisystem, multifocal bone is effective, RR good Age >2 yr at 2-CdA |
| LCH-S-2005 | RO− 62% (RR) | RO+ 48% | ||
| RO+ (n=46) | RO− 97% | |||
| RO− (n=37) | ||||
| 2-CdA/Ara-C (9 mg/m2, 1g/m2/d×5 d) [34] | Prospective | RR 92% | 5yr-OS 85% | 2-CdA/Ara-C is effective for RO+ multisystem LCH |
| LCH-S-2005 | No active: n=2, Better: 23, | High toxicity (grade 4 hematologic toxicity, severe infection) | ||
| (n=27, RO+) | Stable: 2 | |||
| Clofarabine (25 mg/m2/d×5 d) [35] | Retrospective | CR (61%) | 1-yr PFS 76% | All patients developed grade 4 neutropenia |
| LCH (n=11), | PR (22%) | 1-yr OS 91% | ||
| RO+ (n=3) | ||||
| RO− (n=8) | ||||
| HSCT | ||||
| MAC vs. RIC (CIBMTR & EBMT) [36] | Retrospective (after 2000) | Relapse rate | 3-yr OS | OS, PFS of MAC and RIC similar. |
| MAC (n=41) vs. RIC (n=26) | MAC (8%) vs. RIC (28%) | MAC 77% | Relapse rate after RIC marginally higher MAC | |
| RIC 71% | ||||
| MAC vs. RIC (Japan) [37] | Retrospective | OS | ||
| MAC (n=11) vs. RIC (n=19) | MAC 63.6% | |||
| RIC 56.8% | ||||
| FFS | ||||
| MAC 54.6% | ||||
| RIC 56.8% | ||||
CIBMTR, center for international blood and marrow transplant research; CR, complete response; EBMT, european blood and marrow transplant; MAC, myeloablative conditioning; OS, overall survival; PFS, progression free survival; PR, partial response; RIC, reduced intensity conditioning; RR, response rate; RO, risk organ.