Rationale for HSCT

Source: Pediatric Hematopoietic Stem Cell Transplant and Cellular Therapy for Cancer (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: June 13, 2024 · Captured 2026-09-09.

Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary. Independent clinical review is pending.

Context: General Information About Hematopoietic Stem Cell Transplant (HSCT)

Blood and marrow transplant, or HSCT, is a procedure that involves infusion of hematopoietic stem cells (along with hematopoietic progenitor cells) to reconstitute the hematopoietic system of a patient. The infusion of hematopoietic stem cells generally follows a preparative regimen consisting of agents designed to do the following:

Create marrow space.

Suppress the patient's immune system to prevent rejection.

Eradicate malignant cells in patients with cancer.

HSCT is currently used in the:

Treatment of malignancies,

Replacement or modulation of an absent or poorly functioning hematopoietic or immune system, or for the

Treatment of certain genetic diseases. In these cases, insufficient expression of the affected gene product can be partially or completely overcome by circulating hematopoietic stem cells transplanted from a donor with normal gene expression.

This summary focuses on the use of HSCT in the treatment of childhood malignancies.

Preserved source evidence · Independent clinical review pending · Not medical advice