MSCs may be useful for treating established severe GVHD after stem cell transplantation. Preliminary data on 40 patients with steroid refractory grade III-IV acute GvHD were recently presented (K Leblanc
Conditions
Interventions
Product Name: mesenchyal stromal cells
Product Code: MSC
Pharmaceutical Form:
Sponsors
University medical Center Utrecht
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. For screening and MSC culture: newly diagnosed acute grade II-IV GVHD or chronic GVHD with an acute pattern matching grade II-IV after allogeneic stem cell transplantation 2. For infusion: steroid refractory grade II-IV acute GVHD or chronic GVHD with an acute pattern matching grade II-IV after allogeneic stem cell transplantation. 3. Patients must have received 2 mg/kg/day of prednisolon for at least 3 consecutive days and experience progression of GVHD or no response to at least 7 days of steroid treatment. 4. In addition to steroids the patient has received either cyclosporin (trough level 150-250 ng/ml) or tacrolimus (trough level 5-15 ng/ml). Patients with steroid refractory GvHD may be treated with second line therapy according to the clinical practice at the center while waiting for MSC infusion. At each center all patients included in the study should be treated in the same way. 5. Written informed consent. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients with poor performance, not expected to survive 3 weeks. 2. Donor Chimerism below 90% 3. Active uncontrolled CMV, EBV or fungal infection
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate safety, toxicity and feasibility of MSC infusion using a platelet lysate expansion method, for the treatment of acute steroid refractory Graft versus Host disease;Secondary Objective: To evaluate efficacy of MSC for the treatment of GVHD, Incidence of infections, Morbidity and mortality of patients treated with MSC, Disease relapse, Immune function and chimerism ;Primary end point(s): Primary endpoint: Response of acute GVHD Response is defined as complete or partial resolution of acute GvHD. Assessment of GVHD response as per protocol 1. Complete resolution of GVHD: Control of all signs and symptoms attributed to acute GVHD (consistent with overall grade 0) for at least one month, with or without continued medication with steroids and cyclosporine 2. Partial resolution of GVHD: Control of some signs and symptoms attributed to acute GVHD with an improvement of overall grade by at least two grades for less than one month. 3. Refractory GVHD: No change in signs or symptoms of GVHD within 8 days of MSC infusion 4. Worsening GVHD: Any progress of GVHD signs and symptoms that increase overall grade by one or more grades 8 days after MSC infusion. 1. and 2. will be defined as response. 3. and 4. will be defined as no response. Evaluation of secondary outcome variables 1. Determination of incidence of chronic GVHD. 2. Survival. 3. Infections. 4. Recurrent disease. | — |
Countries
Netherlands
Outcome results
None listed