Clinical trial NCT04580576: Gender Differences in Pediatric Hematopoietic Stem Cell Transplantation (HSCT)
Official title: Pilot Study on Gender Differences in Hematopoietic Cell Transplantation Outcomes in the Pediatric Population
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hematopoietic Stem Cell Transplantation
Keywords: gender, pediatrics, hematopoietic cell transplantation
Lead sponsor: IRCCS Burlo Garofolo
Sponsor class: OTHER
First posted: 2020-10-08
Enrollment: 200
Sex: ALL
Minimum age (years): 4
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Population of pediatric patients undergoing hematopoietic stem cell transplantation

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.


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

Primary outcomes:
measure: Gender-related difference in overall 12-month toxicity
timeFrame: 12 months after transplant
Secondary outcomes:
measure: Gender difference in overall survival (OS)
timeFrame: 12 months after transplant
measure: Gender difference in post-transplant primary disease recurrence
timeFrame: 12 months after transplant
measure: Gender difference in the frequency of transplant-related toxicity at 12 months
timeFrame: 12 months after transplant
measure: Gender difference in infectious complications
timeFrame: 12 months after transplant
measure: Gender difference in the frequency of adverse events due to pre-transplant conditioning regimen
timeFrame: 12 months after transplant
measure: Gender difference in severity of adverse events due to pre-transplant conditioning regimen
timeFrame: 12 months after transplant
measure: Gender difference in timing of hematological engraftment
timeFrame: 12 months after transplant
measure: Gender difference in frequency of primary graft failure
timeFrame: 12 months after transplant
measure: Gender difference in frequency of secondary graft failure
timeFrame: 12 months after transplant
Other outcomes:
Locations:
Study officials:
affiliation: Institute for Maternal and Child Health IRCCS Burlo Garofolo
name: Alessandra Maestro, PharmD, PhD
role: STUDY_DIRECTOR
affiliation: Institute for Maternal and Child Health IRCCS Burlo Garofolo
name: Natalia Maximova, MD
role: PRINCIPAL_INVESTIGATOR
References: