Patients with hematological malignancies are at high risk of infections due to both the disease and the associated treatments. The use of immunoglobulin (Ig) to prevent infections is increasing in this population, but its cost effectiveness is unknown. This trial-based economic evaluation aimed to compare the cost effectiveness of prophylactic Ig with prophylactic antibiotics in patients with hematological malignancies.

The economic evaluation used individual patient data from the RATIONAL feasibility trial, which randomly assigned 63 adults with chronic lymphocytic leukemia, multiple myeloma, or lymphoma to prophylactic Ig or prophylactic antibiotics. The following two analyses were conducted to estimate the cost effectiveness of the two treatments over the 12-month trial period from the perspective of the Australian health system:

(i) a cost-utility analysis (CUA) to assess the incremental cost per quality-adjusted life-year (QALY) gained using data collected with the EuroQol 5D-5L questionnaire; and

(ii) a cost-effectiveness analysis (CEA) to assess the incremental cost per serious infection prevented (grade ≥3) and per infection prevented (any grade).

The total cost per patient was significantly higher in the Ig arm than in the antibiotic arm (difference AUD29,140 [USD19,000]). There were non-significant differences in health outcomes between the treatment arms: patients treated with Ig had fewer QALYs (difference −0.072) and serious infections (difference −0.26) than those given antibiotics, but more overall infections (difference 0.76). The incremental cost-effectiveness from the CUA indicated that Ig was more costly than antibiotics and associated with fewer QALYs. In the CEA, Ig costed an additional AUD111,262 (USD73,000) per serious infection prevented, but it was more costly than antibiotics and associated with more infections when all infections were included.

These results indicate that, on average, Ig prophylactic treatment may not be cost effective compared with prophylactic antibiotics for the group of patients with hematological malignancies recruited to the RATIONAL feasibility trial. Further research is needed to confirm these findings in a larger population and over the longer term.

Patients with hematological malignancies are likely to develop hypogammaglobulinemia. Immunoglobulin (Ig) is commonly given to prevent infections, but its overall costs and cost-effectiveness are unknown.

A systematic review was conducted following the PRISMA guidelines to assess the evidence on the costs and cost-effectiveness of Ig, administered intravenously (IVIg) or subcutaneously (SCIg), in adults with hematological malignancies.

Six studies met the inclusion criteria, and only two economic evaluations were identified; one cost-utility analysis (CUA) of IVIg versus no Ig, and another comparing IVIg with SCIg. The quality of the evidence was low. Compared to no treatment, Ig reduced hospitalization rates. One study reported no significant change in hospitalizations following a program to reduce IVIg use, and an observational study comparing IVIg with SCIg suggested that there were more hospitalizations with SCIg but lower overall costs per patient. The CUA comparing IVIg versus no Ig suggested that IVIg treatment was not cost-effective, and the other CUA comparing IVIg to SCIg found that home-based SCIg was more cost-effective than IVIg, but both studies had serious limitations.

Our review highlighted key gaps in the literature: the cost-effectiveness of Ig in patients with hematological malignancies is very uncertain. Despite increasing Ig use worldwide, there are limited data regarding the total direct and indirect costs of treatment, and the optimal use of Ig and downstream implications for healthcare resource use and costs remain unclear. Given the paucity of evidence on the costs and cost-effectiveness of Ig treatment in this population, further health economic research is warranted.

Patients with hematological malignancies are likely to develop hypogammaglobulinemia (HGG) and subsequent infections. Immunoglobulin (Ig) replacement is commonly given to prevent infections, but the total costs and cost effectiveness of its use are unknown.

A systematic review was conducted following PRISMA guidelines to assess evidence on the costs and cost effectiveness of Ig replacement, administered intravenously (IVIg) or subcutaneously (SCIg), in adult patients with hematological malignancies. This review was registered with PROSPERO (CRD42022321908).

Six studies were included out of a total of 3,612 citations. A narrative synthesis was conducted because of the high level of heterogeneity across the included studies. Two economic evaluations were identified: one cost-utility analysis (CUA) of IVIg versus no Ig and one comparing IVIg with SCIg. The quality of the evidence was low, with most studies having small patient numbers and a high risk of bias. Compared with no treatment, Ig replacement reduced the hospitalization rate in patients with hematological malignancies.

One study reported no change in hospitalization rates following a program to reduce IVIg use, and an observational study comparing IVIg with SCIg found more hospitalizations with SCIg but lower total costs per patient. The CUA comparing IVIg with no IVIg suggested that IVIg treatment was not cost effective, but this study was published in 1991 and had significant limitations. The other CUA found that home-based SCIg was more cost effective than IVIg, but model inputs were derived from unpublished data in a very small patient cohort with HGG and different malignancies.

Our review highlights key gaps in the literature. The cost effectiveness of Ig replacement in patients with hematological malignancies is still very uncertain. Despite the increasing use of Ig replacement there are limited data regarding its direct and indirect costs, and its optimal use and implications for healthcare resources remain unclear. Given the paucity of data on the cost and cost effectiveness of Ig treatment in this population, further health economic research is warranted.