Gender Differences in Pediatric Hematopoietic Stem Cell Transplantation (HSCT)

NCT04580576 | Completed

• 1 other identifier: RC 25/2019
• Study Type: observational
• Target: 200
• Locations: 0 countries
• Sites: N/A

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

Keywords

gender, pediatrics, hematopoietic cell transplantation

Outcome Measures

Primary Outcomes (1)

• Gender-related difference in overall 12-month toxicity

  Differences in toxicity (hepatic, renal, pulmonary, gastrointestinal) in males and females recipients

  12 months after transplant

Secondary Outcomes (9)

• Gender difference in overall survival (OS)

  12 months after transplant

• Gender difference in post-transplant primary disease recurrence

  12 months after transplant

• Gender difference in the frequency of transplant-related toxicity at 12 months

  12 months after transplant

• Gender difference in infectious complications

  12 months after transplant

• Gender difference in the frequency of adverse events due to pre-transplant conditioning regimen

  12 months after transplant

Study Arms (2)

Female group

Pediatric female patients undergoing hematopoietic stem cell transplantation

Procedure: Hematopoietic stem cell transplantation

Male group

Pediatric male patients undergoing hematopoietic stem cell transplantation

Procedure: Hematopoietic stem cell transplantation

Interventions

Hematopoietic stem cell transplantation PROCEDURE

Allogeneic or autologous bone marrow transplant

Female group; Male group

Eligibility Criteria

• Age: 4 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Population of pediatric patients undergoing hematopoietic stem cell transplantation

You may qualify if:

• Patients aged between 4 months and 17 years
• Diagnosis of oncohaematological disease subjected to hematopoietic stem cell transplantation
• Allogeneic or autologous bone marrow transplantation from January 2000 to October 2018
• Consent acquired for the processing of data for research purposes

Sponsors & Collaborators

• IRCCS Burlo Garofolo: lead

MeSH Terms

Conditions

Coitus

Interventions

Hematopoietic Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Sexual Behavior; Behavior

Intervention Hierarchy (Ancestors)

Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Alessandra Maestro, PharmD, PhD

  Institute for Maternal and Child Health IRCCS Burlo Garofolo

  STUDY DIRECTOR

• Natalia Maximova, MD

  Institute for Maternal and Child Health IRCCS Burlo Garofolo

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 2, 2020
• First Posted: October 8, 2020
• Study Start: January 1, 2000
• Primary Completion: October 1, 2018
• Study Completion: October 1, 2019
• Last Updated: October 8, 2020

Record last verified: 2020-10

Data Sharing

• IPD Sharing: Will not share