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Related Concept Videos

Randomized Experiments01:13

Randomized Experiments

The randomization process involves assigning study participants randomly to experimental or control groups based on their probability of being equally assigned. Randomization is meant to eliminate selection bias and balance known and unknown confounding factors so that the control group is similar to the treatment group as much as possible. A computer program and a random number generator can be used to assign participants to groups in a way that minimizes bias.
Simple randomization
Simple...
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...
Tissue Transplantation01:24

Tissue Transplantation

Tissue transplantation is a significant medical procedure involving the transfer of cells, tissues, or organs from a donor to a recipient, with the primary aim of restoring lost functions. This procedure is crucial in treating a broad spectrum of diseases, including kidney diseases, liver failure, heart disease, and certain types of cancers.
The Biology of Tissue Transplantation
The biology of tissue transplantation hinges on the Major Histocompatibility Complex (MHC) molecules. These molecules...
Regulation of Hematopoietic Stem Cells01:01

Regulation of Hematopoietic Stem Cells

All blood and immune cells are produced from the multipotent hematopoietic stem cells (HSCs) by the process of hematopoiesis. However, they all have a limited life span. In addition, many are depleted in immune surveillance or combatting an injury or infection. This makes blood one of the most regenerative tissues. Hematopoiesis helps replenish these blood and immune cells, restoring the body's normal functioning. However, overproduction of blood and immune cells can make them cancerous or...
Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches01:23

Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches

Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast, controlled...

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Competitive Transplants to Evaluate Hematopoietic Stem Cell Fitness
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Published on: August 31, 2016

Randomized Interventional Effects in Semicompeting Risks, With Application to a Hematopoietic Cell Transplantation

Yuhao Deng1, Rui Wang2, Tao Zhang3

  • 1Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Center, Seattle, Washington, USA.

Statistics in Medicine
|June 2, 2026
PubMed
Summary
This summary is machine-generated.

This study introduces a method to analyze treatment effects on terminal events mediated by intermediate events, accounting for censoring. Matched unrelated donor transplantation improves survival rates in lymphoma patients post-hematopoietic cell transplantation.

Keywords:
causal inferencecumulative incidence functionhazardmediation analysistime‐to‐event datatime‐varying confounding

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Area of Science:

  • Biostatistics
  • Clinical Epidemiology
  • Survival Analysis

Background:

  • Intermediate events can modify the risk of primary (terminal) events, creating semicompeting risks in clinical studies.
  • Decomposing treatment effects into direct and indirect pathways is crucial for understanding mediated effects.

Purpose of the Study:

  • To extend the randomized interventional approach to time-to-event outcomes with intermediate and terminal events under right censoring.
  • To develop methods for identifying and estimating direct and indirect treatment effects in the presence of semicompeting risks.

Main Methods:

  • Utilizing a randomized interventional approach with a random draw for the intermediate event process.
  • Presenting an identification formula for interventional effects and discussing assumption variants.
  • Employing nonparametric maximum likelihood estimation and proposing a latent frailty sensitivity analysis.

Main Results:

  • The study illustrates the method using hematopoietic cell transplantation data, analyzing death mediated by relapse.
  • Graft-versus-host disease (GVHD) was considered as a time-varying confounder.
  • Matched unrelated donor transplantation showed preferable survival rates compared to haploidentical donor transplantation for lymphoma patients, particularly with post-transplant cyclophosphamide (PTCy) GVHD prophylaxis.

Conclusions:

  • The proposed method effectively decomposes treatment effects in semicompeting risks scenarios.
  • Matched unrelated donor transplantation is a favorable strategy for improving survival in specific lymphoma patient populations undergoing hematopoietic cell transplantation.
  • The findings highlight the importance of considering intermediate events and time-varying confounders in survival analysis.