Trial reportBlood advances2026
Clofarabine for relapsed/refractory LCH and non-LC histiocytosis.
Trial report in Blood advances, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02425904 (Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related Disorders), which is not on this map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase II Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related Disorders
Who cites it
0 citing papers in PubMed.
No citing paper in PubMed yet.
Corrections and comments
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Authors and funding
18 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
abstractLangerhans cell histiocytosis (LCH) and non-Langerhans cell (non-LC) histiocytosis are rare neoplastic diseases with variable behavior. Although chemotherapy is often effective, some patients respond poorly to treatment, experience disease recurrence and/or develop long-term complications. Clofarabine (Clolar), a deoxyadenosine analog, has shown encouraging activity in histiocytic disorders. Here, we report a prospective, multicenter, phase 2 study evaluating the efficacy and toxicity of clofarabine in recurrent/refractory (R/R) LCH (stratum 1) and non-LC histiocytosis (stratum 2). All participants received clofarabine on days 1 through 5 of each 28-day cycle. Response was assessed after 2 cycles; participants without evidence of disease progression received 4 additional cycles. A total of 25 participants enrolled: 20 in stratum 1 and 5 in stratum 2. In stratum 1, 17 of 20 participants (85%; 95% confidence interval [CI], 62%-97%) were responders, and 3 had stable disease after cycle 2. Overall, 19 stratum-1 participants completed 6 cycles; 1 withdrew after cycle 3. No stratum-1 participants progressed during treatment and 3 had disease recurrence after cycle 6, resulting in 2-year progression-free survival ± standard error and overall survival of 89% ± 7% and 100%, respectively (n = 20; median follow-up, 40.9 months [range, 2.7-49.4]). In stratum 2, 3 of 4 evaluable participants (75%; 95% CI, 19%-99%) had partial response. Overall, 20 of 25 (80%) of participants had ≥1 grade ≥3 toxicity attributed to protocol therapy. In conclusion, clofarabine is active and tolerable in children with R/R LCH and warrants further study in patients with non-LC histiocytosis. Further study of clofarabine in these populations is warranted. This trial was registered at www.clinicaltrials.gov (#NCT02425904).
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