Tacrolimus in Children With Henoch-Schönlein Purpura Nephritis

Study Overview
=================
Brief Summary
-----------------
Henoch-Schönlein purpura (HSP) is the most common vasculitis in children, with an incidence of approximately 10:100 000 children and a slight male predominance (male-to-female ratio of 1.5:1). Henoch-Schönlein purpura nephritis (HSPN) is the principal cause of morbidity for HSP and 1%-7% of HSPN patients may progress to renal failure or end-stage renal disease. Immunosuppressive therapy has become the standard treatment in children with HSPN, however the use of these drugs are still mainly in an off-label manner in clinical practice. Tacrolimus, a calcineurin inhibitor, has been recently suggested in the treatment of HSPN in children. However, the evidence-based clinical data are still limited. Given the potential benefits and unmet need in clinical practice, the purposes of this pilot study were to assess effectiveness and safety of tacrolimus in HSPN children and evaluate the potential impact of CYP3A5.

Official Title
-----------------
Off-label Use of Tacrolimus in Children With Henoch-Schönlein Purpura Nephritis: Effectiveness and Safety

Conditions
-----------------
Henoch-Schönlein Purpura Nephritis

Intervention / Treatment
-----------------
* Drug: tacrolimus
* Drug: prednisone


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: HSPN children Aged less than 18 years; receiving tacrolimus as initial immunosuppressive therapy - Exclusion Criteria: Children received other immunosuppressive drug before the trial or other systemic trial drug therapy; Children had a concomitant medical condition, whose participation, in the opinion of the Investigator and/or medical advisor, may create an unacceptable additional risk. -

Ages Eligible for Study
-----------------
Maximum Age: 18 Years

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

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

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

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: therapy group<br>Immunosuppressive therapy included tacrolimus and prednisone. | Drug: tacrolimus<br>* Immunosuppressive therapy included tacrolimus and prednisone. Tacrolimus treatment was initiated at a dosage of 0.05-0.1 mg/kg/day twice daily and used for at least 6 month. Prednisone was started at 2 mg/kg/day and tapered off gradually after initiation of treatment.<br>Drug: prednisone<br>* Immunosuppressive therapy included tacrolimus and prednisone. Tacrolimus treatment was initiated at a dosage of 0.05-0.1 mg/kg/day twice daily and used for at least 6 month. Prednisone was started at 2 mg/kg/day and tapered off gradually after initiation of treatment.<br>|

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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Complete remission | clinical symptoms and signs disappeared and proteinuria was less than 4mg/h per m2 body surface area within 6 months. | within 6 months | 
| Partial remission | if proteinuria was reduced to 4.1-40mg/h per m2 body surface area within 6 months. A nonresponsive patient was defined if there was no improvement in clinical symptoms or signs 6 months after the therapy of tacrolimus with or without prednisone, or urinary protein remained more than 40mg/h per m2 body surface area. | within 6 months | 
| nonresponsive | A nonresponsive patient was defined if there was no improvement in clinical symptoms or signs 6 months after the therapy of tacrolimus with or without prednisone, or urinary protein remained more than 40mg/h per m2 body surface area. | within 6 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
tacrolimus, Henoch-Schönlein purpura nephritis, children