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Efficacy and Tolerance of Cladribine for Non-Langerhans Cell Histiocytosis
Quentin Riller1, Thouraya Ben Salem1, Jean-François Emile2
1Sorbonne Université, Assistance Publique-Hôpitaux De Paris, Service de Médecine Interne 2, Centre National De Référence Maladies Systémiques Rares et Histiocytoses, Hôpital Pitié-Salpêtrière, Centre D'immunologie et Des Maladies Infectieuses, INSERM-CNRS U1135, Paris, France.
Insights
Cladribine shows efficacy in treating rare non-Langerhans cell histiocytoses like Erdheim-Chester Disease (ECD) and Rosai-Dorfman Disease (RDD). The drug demonstrated a 62% clinical response rate, with a manageable safety profile, offering new treatment options for these conditions.
Area of Science:
- Hematology
- Oncology
- Rare Diseases
Background:
- Erdheim-Chester Disease (ECD) and Rosai-Dorfman Disease (RDD) are rare non-Langerhans cell histiocytoses.
- These conditions share features including CD1a- histiocyte accumulation.
- Cladribine's efficacy in Langerhans-cell histiocytoses is known; its use in non-Langerhans forms was undetermined.
Purpose of the Study:
- To assess the efficacy of cladribine in patients with ECD, RDD, or non-classified non-Langerhans cell histiocytosis.
- To evaluate clinical and radiological responses to cladribine treatment.
- To analyze the safety profile of cladribine in this patient population.
Main Methods:
- Retrospective assessment of cladribine efficacy.
- Inclusion of consecutive patients diagnosed with ECD, RDD, or non-classified non-Langerhans cell histiocytosis.
- Evaluation based on clinical and radiological response criteria.
Main Results:
- A clinical overall response rate (ORR) of 62% was observed (44% in ECD, 70% in RDD).
- Radiological ORR was 43% (44% in ECD, 30% in RDD).
- Favorable responses were noted in cranial nerve palsy cases, while CNS involvement showed limited improvement. Lymphopenia was common, but infectious events were rare.
Conclusions:
- Cladribine demonstrates efficacy in treating non-Langerhans cell histiocytoses.
- The study provides new data on the safety and effectiveness of cladribine for ECD and RDD.
- Cladribine represents a potential therapeutic option for these rare histiocytic disorders.
Background:
Erdheim-Chester Disease (ECD) and Rosai-Dorfman Disease (RDD) Are Rare Non-Langerhans Cell Histiocytoses That Share Several Clinical and Histological Features, Including the Accumulation of CD1a- Histiocytes in Organs. Cladribine, a Purine Analog, Leads to an Overall Response Rate (ORR) of 91% in Langerhans-Cell Histiocytoses. Whether the Same Results Could Be Obtained in Non-Langerhans Cell Histiocytoses Remains To Be Determined.
Patients And Methods:
We retrospectively assessed the efficacy of cladribine according to clinical and radiological responses in consecutive patients with a diagnosis of ECD, RDD, or non-classified non-Langerhans cell histiocytosis.
Results:
Twenty-One Patients Were Included in This Study (17 Males, Median Age at Cladribine Treatment 53 Years). The Clinical ORR Was 62% (44% in ECD, 70% in RDD), whereas the Radiological ORR Was 43% (44% in ECD, 30% in RDD). Four of Five Patients With Cranial Nerve Palsy Responded Clinically (80%), whereas Pseudo-Degenerative CNS Involvement Did Not Improve (n = 3). Six Patients With Multisystemic Involvement Did Not Require Additional Treatment After Achieving a Radiological Response (n = 4) or After Achieving Radiological Stable Disease (n = 2), with a Median Follow-Up of 2.3 Years (Range 0.5-9.5). After They Achieved a Radiological Response, 4/9 (44%) Patients Relapsed in a Median Time of 18 Months (Range 6-95). The Safety Profile Showed That 19/19 Patients Experienced Lymphopenia, Whereas Only 2/19 Had Clinical Infectious Events (9%).
Conclusions:
These Results Provide New Evidence of the Efficacy of Cladribine in Non-Langerhans Cell Histiocytosis and Brings New Data on the Safety Profile of This Drug in Histiocytoses.
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