Costs and cost-effectiveness of allogeneic stem cell transplantation in children are predictable

Susanne Matthes-Martin1, Ulrike Pötschger, Ronald Barr

  • 1Department of Pediatrics, St Anna Children's Hospital, Medical University of Vienna, Vienna, Austria. susanne.matthes@stanna.at

Insights

The total cost of pediatric stem cell transplantation (SCT) varies widely, influenced by patient age, disease status, and donor type. Despite high costs, SCT remains cost-effective for life-threatening conditions.

Area of Science:

  • Pediatric Hematology/Oncology
  • Health Economics
  • Transplantation Medicine

Background:

  • Pediatric stem cell transplantation (SCT) is a critical treatment for various life-threatening conditions.
  • Understanding the economic implications of SCT is essential for resource allocation and healthcare policy.
  • Previous cost analyses for pediatric SCT are limited, particularly regarding comprehensive cost-effectiveness evaluations.

Purpose of the Study:

  • To determine the overall costs associated with pediatric stem cell transplantation (SCT), including donor search and first-year post-transplant expenses.
  • To analyze the correlation between SCT costs and patient/transplantation characteristics, as well as a risk score for transplantation-related mortality.
  • To evaluate the cost-effectiveness of pediatric SCT, calculating costs per surviving patient and per life-year gained.

Main Methods:

  • A cohort of 141 pediatric patients undergoing SCT at a single institution was analyzed.
  • Costs were systematically collected, encompassing donor search and the first year post-SCT.
  • Statistical analyses correlated costs with patient demographics, disease status, donor type, and a validated transplantation-related mortality risk score.

Main Results:

  • The median overall cost for pediatric SCT was €136,382, with significant variability (€26,897 to €601,348).
  • Increased costs were associated with older patient age, use of non-matched sibling donors, and advanced disease.
  • Costs strongly correlated with the transplantation-related mortality risk score, rising from €89,550 (score 0) to €274,915 (score 3).
  • Cost-effectiveness decreased with higher risk scores, with costs per survivor ranging from €93,209 to €1,216,348.

Conclusions:

  • Pediatric SCT costs exhibit substantial variation, predictable by patient age, disease, and donor type.
  • The cost-effectiveness of pediatric SCT is within acceptable ranges for severe hemato-oncologic diseases, even for high-risk patients.
  • These findings provide valuable economic data for healthcare providers and policymakers involved in pediatric transplantation.

Related Concept Videos

Bone Marrow Sampling and Transplants01:22

Bone Marrow Sampling and Transplants

Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy the...
Stem Cell Culture01:17

Stem Cell Culture

Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
Stem Cell Therapy for Tissue Regeneration01:21

Stem Cell Therapy for Tissue Regeneration

Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
Induced Pluripotent Stem Cells01:06

Induced Pluripotent Stem Cells

Stem cells are undifferentiated cells that divide and produce different cell types. Ordinarily, cells that have differentiated into a specific cell type are terminally differentiated; however, scientists have found a way to reprogram these mature cells so that they dedifferentiate and return to an unspecialized, proliferative state. These cells are pluripotent like embryonic stem cells—able to produce all cell types—and are called induced pluripotent stem cells (iPSCs).
Somatic cells are...