title: The potential for treatment of idiopathic thrombocytopenic purpura with anti-D to prevent splenectomy: a predictive cost analysis.
abstract: Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disorder affecting both children and adults that can be manifested by severe bleeding episodes. Adult ITP patients have a low rate of spontaneous remission, and symptomatic patients commonly undergo splenectomy; however, maintenance therapy may increase the rate of remission, allowing splenectomy to be avoided. Anti-D is a recently licensed treatment for ITP that has the potential to delay, and possibly avoid, the need for splenectomy. We used preliminary data from an ongoing clinical trial to evaluate the costs involved in using anti-D therapy for 1 year with the intent of avoiding the need for splenectomy. We accounted for different possible outcomes at the completion of the clinical trial. An economic model with a theoretical cohort of 100 patients was developed using the model of an ongoing clinical trial. The average wholesale price was used to determine the cost of an infusion of anti-D based on an average dose ($1,213 per infusion). The cost of splenectomy was determined by a literature review ($16,000). Costs were calculated for all known patient outcomes; where outcomes were unknown and likely to vary, all possible outcomes were accounted for (splenectomy or no splenectomy). In our theoretical cohort, 31 of 100 patients were taken off anti-D and received splenectomy, 32 of 100 were stable after receiving anti-D and would not need splenectomy, and 37 of 100 had Indeterminate outcomes after receiving anti-D. When compared with the cost of the hypothetical scenario of initially giving all 100 patients splenectomy ($1.6 million), a minimum of 47 patients would have to avoid splenectomy to result in a cost savings for our cohort of 100 patients. The group of 47 patients avoiding splenectomy would be composed of the 32 patients comprising the stable group and at least 15 of the 37 patients comprising the group with indeterminate outcomes. If all 37 of the patients in the group with indeterminate outcomes avoid splenectomy, $363,000 and 69 spleens would be saved. Our data suggest that in the phase III trial of maintenance anti-D therapy versus immediate splenectomy, anti-D therapy will be a cost-effective option if 47% or more of patients avoid splenectomy.
authors.affiliation: Division of Hematology/Oncology and General Internal Medicine, Northwestern University Medical School, Chicago, IL 60611, USA.
authors.forename: C L
authors.initials: CL
authors.lastname: Bennett
authors.forename: P D
authors.initials: PD
authors.lastname: Weinberg
authors.forename: R M
authors.initials: RM
authors.lastname: Golub
authors.forename: J B
authors.initials: JB
authors.lastname: Bussel
mesh_terms: D000328:Adult; D003365:Costs and Cost Analysis; D006801:Humans; D007518:Isoantibodies / Q000008:administration & dosage / Q000191:economics* / Q000627:therapeutic use; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy / Q000191:economics* / Q000601:surgery; D018029:Rho(D) Immune Globulin; D013156:Splenectomy / Q000191:economics*
chemical_list: D007518:Isoantibodies; C061961:RHO(D) antibody; D018029:Rho(D) Immune Globulin