Gender Differences in Pediatric Hematopoietic Stem Cell Transplantation (HSCT)

Study Overview
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Brief Summary
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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.

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

Conditions
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Hematopoietic Stem Cell Transplantation

Intervention / Treatment
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* Procedure: Hematopoietic stem cell transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 4 Months
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Female group<br>Pediatric female patients undergoing hematopoietic stem cell transplantation | Procedure: Hematopoietic stem cell transplantation<br>* Allogeneic or autologous bone marrow transplant<br>|
| Male group<br>Pediatric male patients undergoing hematopoietic stem cell transplantation | Procedure: Hematopoietic stem cell transplantation<br>* Allogeneic or autologous bone marrow transplant<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Gender difference in overall survival (OS) | Overall survival comparison from males and females recipients | 12 months after transplant | 
| Gender difference in post-transplant primary disease recurrence | Incidence of post-transplant leukemic relapse in males and females recipients | 12 months after transplant | 
| 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 | 
| Gender difference in infectious complications | Number of episodes of sepsis / fungal infections / viral reactivations after HSCT | 12 months after transplant | 
| 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 | 
| 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 | 
| 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 | 
| 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 | 
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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gender, pediatrics, hematopoietic cell transplantation