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Response to mercaptopurine for refractory autoimmune cytopenias in children
Amy Sobota1, Ellis J Neufeld, Sameer Lapsia
1Division of Hematology/Oncology, Children's Hospital Boston, Boston, Massachusetts 02115, USA.
Insights
6-Mercaptopurine (6MP) is an effective single-agent treatment for pediatric refractory immune cytopenias like ITP. This study found an 83% response rate, suggesting 6MP as a viable option for children unresponsive to initial therapies.
Area of Science:
- Pediatric Hematology
- Immunology
- Pharmacology
Background:
- Severe immune cytopenias in children, including immune thrombocytopenic purpura (ITP), auto-immune hemolytic anemia (AIHA), and Evans syndrome, often require alternative treatments when initial therapies fail.
- 6-Mercaptopurine (6MP) is a potential therapeutic option, but its use as a single agent in pediatric populations has been limited, with few reported case series since 1970.
Purpose of the Study:
- To evaluate the efficacy and safety of 6-Mercaptopurine (6MP) as a steroid-sparing monotherapy for pediatric patients with refractory immune cytopenias.
- To assess the response rates and identify potential side effects associated with 6MP treatment in this patient group.
Main Methods:
- A retrospective review of 29 pediatric patients treated with 6MP between 2000 and 2007 at a single institution.
- Patients had diagnoses of ITP, AIHA, or Evans syndrome and were refractory to prior treatments.
- Response criteria included a hemoglobin increase of ≥1.5 g/dL to ≥10 g/dL for anemia or a platelet count ≥50 x 10^9/L for thrombocytopenia.
Main Results:
- An overall response rate of 83% was observed across all treated patients.
- Fourteen percent of patients discontinued 6MP due to adverse side effects.
- The treatment demonstrated effectiveness in improving hematological parameters for various immune cytopenias.
Conclusions:
- 6-Mercaptopurine (6MP) shows promise as an effective single-agent treatment for children with refractory immune cytopenias.
- Further prospective studies are necessary to confirm long-term efficacy, assess toxicity profiles, and delineate specific patient subgroups most likely to benefit from 6MP therapy.
Background:
Several treatment strategies are available for children with severe immune thrombocytopenic purpura (ITP) and other immune cytopenias refractory to initial therapies. 6-Mercaptopurine (6MP) is one option, however it has not been well studied in children, especially as a single agent, and no pediatric case series have been reported since 1970.
Patients And Methods:
We reviewed the experience at our institution over 8 years, using 6MP as a steroid sparing treatment for children with ITP, auto-immune hemolytic anemia (AIHA) or Evans syndrome. A total of 29 pediatric patients were treated with 6MP from 2000 to 2007.
Results:
Response was defined as a rise in hemoglobin by at least 1.5 g/dl and to a level of 10 g/dl or greater in patients treated for anemia, or a platelet count >or=50 x 10(9)/L in patients treated for thrombocytopenia. We found an overall response rate of 83% among all patients. Fourteen percent of patients stopped drug because of side effects.
Conclusions:
These results suggest that 6MP can be an effective single-agent treatment for refractory immune cytopenias in children. Prospective studies are warranted to determine long-term efficacy and toxicity and to more clearly define patient populations most likely to respond.
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