Journal
BLOOD
Volume 121, Issue 25, Pages 5006-5014Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2012-09-455774
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Funding
- Children's Cancer Research Institute (Vienna, Austria)
- Anthony House
- Histiocytosis Association of America
- Histiocytosis Association of Belgium
- Histiocytosis Association of Italy
- Histiocytosis Association of Canada
- Histiozytosehilfe (Germany)
- Programme Hospitalier de Recherche Clinique
- CHU, Nantes (France)
- Swedish Childhood Cancer Foundation
- Swedish Research Council
- Children's Research Institute (Washington, DC)
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Langerhans cell histiocytosis (LCH)-III tested risk-adjusted, intensified, longer treatment of multisystem LCH (MS-LCH), for which optimal therapy has been elusive. Stratified by risk organ involvement (high [RO+] or low [RO-] risk groups), >400 patients were randomized. RO+ patients received 1 to 2 six-week courses of vinblastine+prednisone (Arm A) or vinblastine+prednisone+methotrexate (Arm B). Response triggered milder continuation therapy with the same combinations, plus 6-mercaptopurine, for 12 months total treatment. 6/12-week response rates (mean, 71%) and 5-year survival (84%) and reactivation rates (27%) were similar in both arms. Notably, historical comparisons revealed survival superior to that of identically stratified RO+ patients treated for 6 months in predecessor trials LCH-I (62%) or LCH-II (69%, P<.001), and lower 5-year reactivation rates than in LCH-I (55%) or LCH-II (44%, P<.001). ROpatients received vinblastine+prednisone throughout. Response by 6 weeks triggered randomization to 6 or 12 months total treatment. Significantly lower 5-year reactivation rates characterized the 12-month Arm D (37%) compared with 6-month Arm C (54%, P=.03) or to 6-month schedules in LCH-I (52%) and LCH-II (48%, P<.001). Thus, prolonging treatment decreased RO- patient reactivations in LCH-III, and although methotrexate added no benefit, RO+ patient survival and reactivation rates have substantially improved in the 3 sequential trials. (Trial No. NCT00276757 www.ClinicalTrials.gov).
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