Prochymal Expanded Access Treatment for Pediatric Patients Who Have Failed Steroids for Acute GVHD

Expanded Access of Prochymal (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Treatment of Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD

This protocol allows for the treatment of pediatric patients, male and female, between the ages of 2 months and 17 years. Patients must have failed steroid treatment for Grade B-D acute GVHD.

For the treatment of pediatric patients who have failed to respond to steroid treatment for acute graft-versus-host disease (GVHD). Failing steroid treatment for acute GVHD is defined as any Grade B-D (IBMTR grading) of acute GVHD that is not improving after at least 3 days of methylprednisolone (≥ 1 mg/kg/day) or equivalent.

Patients will be treated with Prochymal twice per week at a dose of 2 x 10^6 hematopoietic stem cells (hMSC)/kg (actual body weight) for each of 4 consecutive weeks. Infusions will be administered at least 3 days apart.

Inclusion criteria:

• Patients must sign an informed consent form (ICF) before any protocol-related procedures, including any pre-treatment procedures, are performed.
• Since patients being treated with Prochymal® under this protocol are under 18, a parental signature of informed consent will be required.
• Patients must be 2 months to 17 years of age, inclusive.
• Patients must have failed to respond to steroid treatment for Grades B-D acute GVHD • Failure to respond to steroid treatment for acute GVHD is defined as any Grade B-D acute GVHD that is not improving after at least 3 days of methylprednisolone (≥1 milligram per kilogram per day [mg/kg/day]) or equivalent.

Exclusion criteria:

• Patient must not have a known allergy to bovine or porcine products.
• Patient must not have received a transplant for a solid tumor disease.
• Patients must not have evidence of a pulmonary infiltrate or diffuse alveolar hemorrhage and must be unlikely to require more than 2 liters (L) of oxygen via face mask or an estimated fraction of inspired oxygen (FiO2) of 28% via other delivery methods in order to sustain an O2 saturation of 92% during the next 3 days.