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Response to mercaptopurine for refractory autoimmune cytopenias in children.

ABSTRACT: 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.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.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.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.

SUBMITTER: Sobota A

PROVIDER: S-EPMC2585152 | biostudies-literature | 2009 Jan

REPOSITORIES: biostudies-literature

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Response to mercaptopurine for refractory autoimmune cytopenias in children.

Sobota Amy A Neufeld Ellis J EJ Lapsia Sameer S Bennett Carolyn M CM

Pediatric blood & cancer 20090101 1

<h4>Background</h4>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.<h4>Patients and methods</h4>We reviewed the experience at our institution over 8 years, using 6MP as a steroid sparing treatment for chi ...[more]

PMID: 18726904

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Project description:Background: Autoimmune cytopenias (AICs) are potentially life-threatening complications following hematopoietic cell transplantation (HCT), yet little is understood about the mechanism by which they develop. We hypothesized that discordant B cell and T cell recovery is associated with AICs in transplant patients, and that this might differ based on transplant indication. Methods: In this case control study of children who underwent HCT at our institution, we evaluated the clinical and transplant characteristics of subjects who developed AICs compared to a control group matched by transplant indication and donor type. In cases, we analyzed the state of immune reconstitution, including B cell recovery, T cell recovery, and chimerism, immediately prior to AIC onset. Subjects were stratified by primary indication for transplant as malignancy (n = 7), primary immune deficiency (PID, n = 9) or other non-malignant disease (n = 4). We then described the treatment and outcomes for 20 subjects who developed AICs. Results: In our cohort, cases were older than controls, were more likely to receive a myeloablative conditioning regimen and had a significantly lower prevalence of chronic GVHD. There were distinct differences in the state of immune recovery based on transplant indication. None of the patients (0/7) transplanted for primary malignancy had T cell recovery at AIC onset compared to 71% (5/7) of patients with PID and 33% (1/3) of patients with non-malignant disease. The subset of patients with PID and non-malignant disease who achieved T cell reconstitution (6/6) prior to AIC onset, all demonstrated mixed or split chimerism. Subjects with AIHA or multi-lineage cytopenias had particularly refractory courses with poor treatment response to IVIG, steroids, and rituximab. Conclusions: These results highlight the heterogeneity of AICs in this population and suggest that multiple mechanisms may contribute to the development of post-transplant AICs. Patients with full donor chimerism may have early B cell recovery without proper T cell regulation, while patients with mixed or split donor chimerism may have residual host B or plasma cells making antibodies against donor blood cells. A prospective, multi-center trial is needed to develop personalized treatment approaches that target the immune dysregulation present and improve outcomes in patients with post-transplant AICs.

Dataset Information

Response to mercaptopurine for refractory autoimmune cytopenias in children.

Publications

Response to mercaptopurine for refractory autoimmune cytopenias in children.

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