Prolonged Survival Using Outpatient Palliative Chemotherapy in Two Children With Refractory Acute Myelogenous Leukemia

Abstract

Background: Therapy options for relapsed/refractory acute myelogenous leukemia (AML) are limited. Palliative chemotherapy options have been explored in adult patients, but little evidence exists in children.

Objectives: Describe the clinical course of 2 pediatric patients with refractory AML who transitioned to outpatient palliative chemotherapy with good disease control and quality of life on these regimens.

Patients and methods: Patient 1 was a 2-year-old girl who received a total of 4 cycles of standard chemotherapy with multiple complications and 15% to 20% blasts on marrow subsequent evaluation. An outpatient regimen of decitabine and vorinostat was consequently chosen for her. Patient 2 was a 16-year-old boy with residual disease after induction 1 with arm A with cytarabine, daunorubicin, and etoposide. His induction 2 course was complicated by multiorgan failure secondary to multiple infections including Klebsiella pneumonia and radiographically identified pulmonary fungal disease. On recovery, the marrow showed no disease but after the toxicities of initial therapy, the patient pursued a palliative regimen with azacitidine and lenalidomide.

Results: Patient 1 tolerated her regimen for 14 months, requiring weekly blood products and only one hospitalization for a central-line infection. Her blast count then increased precipitously, the disease progressed, and she died comfortably while receiving hospital-based end-of-life care. Patient 2 tolerated 14 months of his regimen. On a surveillance marrow sample, he was found to have 0.02% minimal residual disease. He then elected to pursue marrow transplantation. He maintained remission until his 6-month posttransplant surveillance bone marrow biopsy, which revealed 0.04% minimal residual disease.

Conclusion: We describe 2 pediatric patients with relapsed/refractory AML who achieved disease control and acceptable quality of life utilizing outpatient palliative chemotherapy for over 12 months. These regimens should be considered in patients who no longer desire cytotoxic chemotherapy or are ineligible for further aggressive approaches.