BACKGROUND: A prospective phase II Histiocyte Society study, LCH-S-98, evaluated the efficacy of 2-chlorodeoxyadenosine (2-CdA) monotherapy as salvage therapy in Langerhans cell histiocytosis (LCH). PROCEDURES: Patients with poor and intermediate risk LCH not responsive to initial therapy and patients with low-risk chronic recurrent LCH were evaluated for response and survival after treatment with 2-6 courses of 2-CdA. RESULTS: Forty-six patients (55%) had involvement of risk organs; lung, liver, spleen, or hematopoetic system (RO+), 37 (45%) were RO-. Twenty-two percent of RO+ patients had a good response while 44% progressed, 62% RO- patients responded, and 11% progressed. Two-year predicted survival is 48% for RO+, 97% for RO- patients, 100% for RO+ patients reactivating in non-risk organs, 67% for RO- patients reactivating in risk organs. Two-year pSU for the entire group is 68%. Seventy-three percent of patients with a poor response to 2-CdA died. Sixty-five percent patients >2 years old and 30% <2 years old survived. There was a median of 26 months from diagnosis to 2-CdA for responders compared to a median of 5 months for non-responders. Twenty-one percent of patients treated <12 months and 57% treated >12 months from diagnosis responded. CONCLUSION: 2-CdA is active in LCH. It produces a higher response rate in patients with low-risk multisystem or multifocal bone disease than those with risk organ involvement. "Risk" patients who fail to respond to 2-CdA have a high mortality. Patient age at 2-CdA therapy and length of time from diagnosis to 2-CdA significantly affect response and survival. (c) 2009 Wiley-Liss, Inc.
BACKGROUND: A prospective phase II Histiocyte Society study, LCH-S-98, evaluated the efficacy of 2-chlorodeoxyadenosine (2-CdA) monotherapy as salvage therapy in Langerhans cell histiocytosis (LCH). PROCEDURES: Patients with poor and intermediate risk LCH not responsive to initial therapy and patients with low-risk chronic recurrent LCH were evaluated for response and survival after treatment with 2-6 courses of 2-CdA. RESULTS: Forty-six patients (55%) had involvement of risk organs; lung, liver, spleen, or hematopoetic system (RO+), 37 (45%) were RO-. Twenty-two percent of RO+ patients had a good response while 44% progressed, 62% RO- patients responded, and 11% progressed. Two-year predicted survival is 48% for RO+, 97% for RO- patients, 100% for RO+ patients reactivating in non-risk organs, 67% for RO- patients reactivating in risk organs. Two-year pSU for the entire group is 68%. Seventy-three percent of patients with a poor response to 2-CdA died. Sixty-five percent patients >2 years old and 30% <2 years old survived. There was a median of 26 months from diagnosis to 2-CdA for responders compared to a median of 5 months for non-responders. Twenty-one percent of patients treated <12 months and 57% treated >12 months from diagnosis responded. CONCLUSION: 2-CdA is active in LCH. It produces a higher response rate in patients with low-risk multisystem or multifocal bone disease than those with risk organ involvement. "Risk" patients who fail to respond to 2-CdA have a high mortality. Patient age at 2-CdA therapy and length of time from diagnosis to 2-CdA significantly affect response and survival. (c) 2009 Wiley-Liss, Inc.
Authors: Stephen J Simko; Benjamin Garmezy; Harshal Abhyankar; Philip J Lupo; Rikhia Chakraborty; Karen Phaik Har Lim; Albert Shih; M John Hicks; Teresa S Wright; Moise L Levy; Kenneth L McClain; Carl E Allen Journal: J Pediatr Date: 2014-10-21 Impact factor: 4.406
Authors: Paul A Veys; Vasanta Nanduri; K Scott Baker; Wensheng He; Giuseppe Bandini; Andrea Biondi; Arnaud Dalissier; Jeffrey H Davis; Gretchen M Eames; R Maarten Egeler; Alexandra H Filipovich; Alain Fischer; Herbert Jürgens; Robert Krance; Edoardo Lanino; Wing H Leung; Susanne Matthes; Gérard Michel; Paul J Orchard; Anna Pieczonka; Olle Ringdén; Paul G Schlegel; Anne Sirvent; Kim Vettenranta; Mary Eapen Journal: Br J Haematol Date: 2015-03-27 Impact factor: 6.998
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