Pro-ADM vs PCT in Patients With Complications Post Hematopoietic Stem Cell Transplantation

Study Overview
=================
Brief Summary
-----------------
Prospective, multicenter, spontaneous, non-interventional, non pharmacological. Study aimed at evaluate kinetics, diagnostic and prognostic value of pro-ADM (proadrenomedullin) as compared to PCT (procalcitonin) in patients presenting with infections or other complications post hematopoietic stem cell transplantation (HSCT)

Detailed Description
-----------------
Evaluation of diagnostic performance of pro-ADM as compared to palliative care therapy (PCT) in paediatric and young adult patients with any type of complications after hematopoietic stem cell transplantation or with transplant related complications.

Official Title
-----------------
Prospective Non-Interventional Trial to Evaluate Kinetics, Diagnostic and Prognostic Value of Pro-ADM as Compared to PCT in Patients Presenting With Infections or Other Complications Post Hematopoietic Stem Cell Transplantation (HSCT)

Conditions
-----------------
Stem Cell Transplant Complications

Intervention / Treatment
-----------------
* Other: observation


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Written informed consent of the parents/legal guardians and patient's assent/consent according to national regulations. For subjects who turn 18 years of age during participation in this study, participation in the future use portion of the study after their 18th birthday requires that they sign the informed consent statement following turning 18 years of age. Age at time of registration from 28 days to less than 39 years of age. Malignant disease (i.e. Acute lymphocytic leukemia (ALL), Acute myeloid leukemia (AML), Myelodysplastic syndromes (MDS)) or nonmalignant disease (e.g. immunodeficiency or inherited disorders of metabolism or hemoglobinopathies etc.) indicated for allogeneic HSCT. Exclusion Criteria: Age less than 28 days Obese patients with body mass index: > 30 kg/m^2. Impaired cardiac function: severe cardiac insufficiency indicated by left ventricle ejection fraction (LVEF) ≤ 35%. Impaired liver function indicated by Bilirubin >3 times the upper limit of normal (ULN), or aspartate aminotransferase/alanine aminotransferase (AST/ALT) >10 times ULN Impaired renal function indicated by estimated glomerular filtration rate (GFR), according to the Schwartz formula for ages 1-17 and according to CDKEPI creatinine equation for ages above 17 years old) < 60 mL/min/1,73m^2. Concurrent severe active infection at the start of conditioning regimen

Ages Eligible for Study
-----------------
Minimum Age: 28 Days
Maximum Age: 39 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Paediatric patients receiving HCTS with infectious complications post HCTS<br>Paediatric patients receiving allogeneic hematopoietic stem cell transplantation (HCTS) and who present with infectious complications post HSCT or with transplant related complications (acute graft-versus-host disease- GvHD-, sinusoidal obstruction syndrome -SOS-, engraftment-ES- and pre-engraftment syndrome- pre-ES-, graft failure, thrombotic microangiopathy associated with HSCT- TA-TMA or those without complications post HSCT). | Other: observation<br>* observation and prospective data collection<br>|
| Paediatric patients receiving HCTS without infectious complications post HCTS<br>Paediatric patients receiving HCTS without infectious complications post HCTS | Other: observation<br>* observation and prospective data collection<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluation of diagnostic performance of pro-ADM as compared to PCT in patients receiving HSCT | In order to achieve the objective, it will be considered paediatric and young adults patients affected by malignant/non-malignant disorders who have been subjected to HSCT and present with infectious complications post HSCT or with transplant related complications (acute graft-versus-host disease (GvHD), sinusoidal obstruction syndrome (SOS), engraftment (ES) and pre-engraftment syndrome (pre-E), graft failure, thrombotic microangiopathy associated with HSCT (TA-TMA) or those without complications post HSCT). | 180 days after HSCT |