Gender Differences in Pediatric Hematopoietic Stem Cell Transplantation (HSCT)

NCT ID: NCT04580576

Last Updated: 2020-10-08

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 200 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2000-01-01
• Study Completion Date: 2019-10-01

Brief Summary

Gender medicine considers the way in which gender, male or female, affects the development and impact of diseases and the response to therapies. It can be said that it is a new transversal dimension of medicine, which evaluates the gender differences in the physiology, pathophysiology and clinic of many diseases and thus sets itself the goal of reaching optimal therapeutic decisions both in men and women based on proven scientific evidence.

Although knowledge of gender medicine has increased significantly in recent years, a gender approach has not been much developed in pediatrics. In the field of bone marrow transplants, hematopoietic stem cell transplantation is known to be the most effective consolidation therapy in some high-risk hematology malignancies such as acute lymphoblastic leukemia and acute myeloid leukemia, and represents one of the potential treatment for patients suffering from solid tumors and genetic hematological, metabolic diseases and primary immunodeficiencies. Huge progress has been made in high resolution donor typing, choice of conditioning regimens, manipulation of hematopoietic stem cells (HSC) and prevention of serious infections in recent years, which have significantly improved the survival rate of patients undergoing to this procedure.

International literature regarding the response and outcomes from hematopoietic cell transplantation in a gender perspective is completely absent, for these reasons this pilot study was born from the need to understand from a broader perspective and in order to better understand how the gender may or not influence the outcome of transplantation in pediatric patients.

This retrospective analysis of the data will concern all patients who underwent allogeneic or autologous bone marrow transplant. The data will be collected from clinical records and from Regional electronic databases. All data will be collected anonymously and an identification code will be assigned to each case.

Conditions

Hematopoietic Stem Cell Transplantation

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

Female group

Pediatric female patients undergoing hematopoietic stem cell transplantation

Hematopoietic stem cell transplantation

Intervention Type: PROCEDURE

Allogeneic or autologous bone marrow transplant

Male group

Pediatric male patients undergoing hematopoietic stem cell transplantation

Hematopoietic stem cell transplantation

Intervention Type: PROCEDURE

Allogeneic or autologous bone marrow transplant

Interventions

Hematopoietic stem cell transplantation

Allogeneic or autologous bone marrow transplant

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

1. Patients aged between 4 months and 17 years

2. Diagnosis of oncohaematological disease subjected to hematopoietic stem cell transplantation

3. Allogeneic or autologous bone marrow transplantation from January 2000 to October 2018

4. Consent acquired for the processing of data for research purposes

• Minimum Eligible Age: 4 Months
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

IRCCS Burlo Garofolo

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alessandra Maestro, PharmD, PhD

Role: STUDY_DIRECTOR

Institute for Maternal and Child Health IRCCS Burlo Garofolo

Natalia Maximova, MD

Role: PRINCIPAL_INVESTIGATOR

Institute for Maternal and Child Health IRCCS Burlo Garofolo

Other Identifiers

RC 25/2019

Identifier Type: -

Identifier Source: org_study_id