We have located links that may give you full text access.
Case Reports
Journal Article
Successful treatment of two children with Langerhans' cell histiocytosis with 2'-deoxycoformycin.
PURPOSE: Langerhans' cell histiocytosis (LCH) is problematic illness with a subset of patients experiencing a relapsing or progressive course despite therapy with chemotherapy and/or immunosuppressive agents. Novel therapies are required. 2'-deoxycoformycin (2'-dCF) is an inhibitor of adenosine deaminase (ADA) and is toxic to lymphocytes and monocytes. We hypothesized that 2'-dCF might be effective in treatment of children with LCH, in part because of recent success reported with 2-chlorodeoxyadenosine. In this report we describe our experience using 2'-dCF to treat two patients with refractory LCH.
PATIENTS AND METHODS: The patients, aged 5 and 3 years, had received therapy for 4 and 2.5 years, respectively, with vinblastine, corticosteroids, etoposide, methotrexate, 6-mercaptopurine, and, in one case, cyclosporine. While receiving cyclosporine, the first patient developed rapidly enlarging lytic skull lesions accompanied by pain and tenderness. While receiving methotrexate and 6-mercaptopurine, the second patient developed fever, anemia, multiple new bony lesions, and massive abdominal and axillary adenopathy, proven at biopsy to be due to recurrent LCH. Treatment in both cases was with 2'-dCF 4 mg/m2 given by intravenous push weekly for 8 weeks then continuing every 2 weeks for >21 and 16 months, respectively.
RESULTS: Both patients responded to 2'-dCF therapy. The first patient's pain resolved following three doses of 2'-dCF, and the skull lesions decreased in size significantly over the following 6 months. After 15 months of therapy he developed asymptomatic radiologic evidence of a possible new lesion in the femur which did not progress over the subsequent 3 months. The second patient experienced improvement at all sites of disease over 8 weeks, with complete resolution of bony lesions over the next 5 months. At 16 months from initiation of 2'-dCF therapy she remains disease-free. Toxicity was limited to asymptomatic grade III-IV lymphopenia and abnormalities of lymphocyte mitogen responses. The patients are asymptomatic and continue biweekly and monthly 2'-dCF therapy, respectively.
CONCLUSION: 2'-dCF has shown activity against LCH in these two cases, with minimal toxicity. Further study of this agent in the therapy of LCH is warranted.
PATIENTS AND METHODS: The patients, aged 5 and 3 years, had received therapy for 4 and 2.5 years, respectively, with vinblastine, corticosteroids, etoposide, methotrexate, 6-mercaptopurine, and, in one case, cyclosporine. While receiving cyclosporine, the first patient developed rapidly enlarging lytic skull lesions accompanied by pain and tenderness. While receiving methotrexate and 6-mercaptopurine, the second patient developed fever, anemia, multiple new bony lesions, and massive abdominal and axillary adenopathy, proven at biopsy to be due to recurrent LCH. Treatment in both cases was with 2'-dCF 4 mg/m2 given by intravenous push weekly for 8 weeks then continuing every 2 weeks for >21 and 16 months, respectively.
RESULTS: Both patients responded to 2'-dCF therapy. The first patient's pain resolved following three doses of 2'-dCF, and the skull lesions decreased in size significantly over the following 6 months. After 15 months of therapy he developed asymptomatic radiologic evidence of a possible new lesion in the femur which did not progress over the subsequent 3 months. The second patient experienced improvement at all sites of disease over 8 weeks, with complete resolution of bony lesions over the next 5 months. At 16 months from initiation of 2'-dCF therapy she remains disease-free. Toxicity was limited to asymptomatic grade III-IV lymphopenia and abnormalities of lymphocyte mitogen responses. The patients are asymptomatic and continue biweekly and monthly 2'-dCF therapy, respectively.
CONCLUSION: 2'-dCF has shown activity against LCH in these two cases, with minimal toxicity. Further study of this agent in the therapy of LCH is warranted.
Full text links
Related Resources
Trending Papers
Challenges in Septic Shock: From New Hemodynamics to Blood Purification Therapies.Journal of Personalized Medicine 2024 Februrary 4
Molecular Targets of Novel Therapeutics for Diabetic Kidney Disease: A New Era of Nephroprotection.International Journal of Molecular Sciences 2024 April 4
The 'Ten Commandments' for the 2023 European Society of Cardiology guidelines for the management of endocarditis.European Heart Journal 2024 April 18
A Guide to the Use of Vasopressors and Inotropes for Patients in Shock.Journal of Intensive Care Medicine 2024 April 14
Get seemless 1-tap access through your institution/university
For the best experience, use the Read mobile app
All material on this website is protected by copyright, Copyright © 1994-2024 by WebMD LLC.
This website also contains material copyrighted by 3rd parties.
By using this service, you agree to our terms of use and privacy policy.
Your Privacy Choices
You can now claim free CME credits for this literature searchClaim now
Get seemless 1-tap access through your institution/university
For the best experience, use the Read mobile app