Patients with hematologic malignancies, particularly those with multiple myeloma, chronic lymphocytic leukemia, and non-Hodgkin lymphoma, are at increased risk for developing hypogammaglobulinemia (HGG) and subsequent infection due to disease-related immune deficiencies or immunosuppression caused by anticancer treatments such as B-cell–targeted therapies and CAR T-cell therapies. Prophylactic immunoglobulin (Ig) is commonly administered in patients with hematologic malignancies to help prevent these events but is more expensive than prophylactic antibiotics. An economic evaluation of Ig vs prophylactic antibiotic treatment for prevention of HGG and infection was conducted using individual patient data from the RATIONAL trial. The RATIONAL trial (N = 63) was a phase II, open-label, multicenter, randomized, controlled feasibility trial in Australia and New Zealand.
In the analysis, most patients in the Ig arm received intravenous Ig. There were no significant differences in healthcare resource use and no significant differences in the mean number of infections and serious infections between the Ig and antibiotic treatment arms. Mean total costs per patient were A$29,140 higher in the Ig arm (A$46,953 vs A$17,813 for antibiotics), mainly due to the higher cost of prophylactic Ig treatment than prophylactic antibiotics. Ig was also associated with fewer quality-adjusted life years (QALYs) than antibiotics.
High level
Over the 12-month trial period, costs were higher for Ig vs prophylactic antibiotic, with no differences in annual infection rates and no significant QALY gains. There is potential for lower incremental costs with more widespread use of subcutaneous Ig, but more research is needed to confirm this. Additional research is also warranted to assess the full health economic impact of Ig. A follow-up trial (ACTRN12622000359730) is currently open for recruitment and aims to determine the full health economic impact of Ig according to HGG severity, previous infections, hematologic diagnoses, and different stages of the treatment pathway, including a larger number of patients and longer follow-up than the phase II RATIONAL trial.
Ground level
In order to achieve a cost-effectiveness benefit for Ig vs prophylactic antibiotic, a very large increase in QALYs and/or a significant improvement in clinical outcomes would be needed, given its high cost. When appropriate, prophylactic antibiotics may be the more cost-effective choice for patients with hematologic malignancies at risk for HGG and infections.
