Gender Differences in Pediatric Hematopoietic Stem Cell Transplantation (HSCT)

Hematopoietic Stem Cell Transplantation Clinical Trial

Official title:

Pilot Study on Gender Differences in Hematopoietic Cell Transplantation Outcomes in the Pediatric Population

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04580576
• Other study ID #: RC 25/2019
• Status: Completed
• Start date: January 1, 2000
• Est. completion date: October 1, 2019

Study information

• Verified date: October 2020
• Source: IRCCS Burlo Garofolo
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 200
• Est. completion date: October 1, 2019
• Est. primary completion date: October 1, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Months to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Hematopoietic Stem Cell Transplantation

Intervention

Procedure:
• Hematopoietic stem cell transplantation
Allogeneic or autologous bone marrow transplant

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
IRCCS Burlo Garofolo

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Gender difference in overall survival (OS)	Overall survival comparison from males and females recipients	12 months after transplant
Secondary	Gender difference in post-transplant primary disease recurrence	Incidence of post-transplant leukemic relapse in males and females recipients	12 months after transplant
Secondary	Gender difference in the frequency of transplant-related toxicity at 12 months	Frequency of post-transplant liver, kidney, pulmonary, gastrointestinal, endocrine, cardiac toxicity	12 months after transplant
Secondary	Gender difference in infectious complications	Number of episodes of sepsis / fungal infections / viral reactivations after HSCT	12 months after transplant
Secondary	Gender difference in the frequency of adverse events due to pre-transplant conditioning regimen	Number of chemo- radiotherapy-related adverse events. Toxicity was graded according to National Cancer Institute (NCI) common toxicity criteria	12 months after transplant
Secondary	Gender difference in severity of adverse events due to pre-transplant conditioning regimen	Severity of chemo- radiotherapy-related adverse events. Toxicity wil be graded according to National Cancer Institute (NCI) common toxicity criteria	12 months after transplant
Secondary	Gender difference in timing of hematological engraftment	Engraftment defined as the engraftment of polymorphonuclear neutrophils (PMN) on the first day of 3 consecutive days with PMN number greater than 500 / ml3 and engraftment of platelets defined as number of platelets> 20,000 / ml3 in the absence of platelet transfusion in the previous 5 days.	12 months after transplant
Secondary	Gender difference in frequency of primary graft failure	Engraftment defined as the engraftment of polymorphonuclear neutrophils (PMN) on the first day of 3 consecutive days with PMN number greater than 500 / ml3 and engraftment of platelets defined as number of platelets> 20,000 / ml3 in the absence of platelet transfusion in the previous 5 days.; Primary graft failure is defined as no evidence of engraftment or hematological recovery of donor cells, within the first month after transplant, without evidence of disease relapse.	12 months after transplant
Secondary	Gender difference in frequency of secondary graft failure	Engraftment defined as the engraftment of polymorphonuclear neutrophils (PMN) on the first day of 3 consecutive days with PMN number greater than 500 / ml3 and engraftment of platelets defined as number of platelets> 20,000 / ml3 in the absence of platelet transfusion in the previous 5 days.; Secondary graft failure refers to the loss of a previously functioning graft, resulting in cytopenia involving at least two blood cell lineages.	12 months after transplant