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Treatment for Steroid Resistent Acute Graft Versus Host Disease

Purpose of study:

Acute GVHD is a serious medical condition that is a common development after Bone Marrow transplant. Acute GVHD happens when the donor cells attack and damage your tissues and organs after transplant. Acute GVHD often causes:

  • Skin rashes
  • Nausea (feeling sick to your stomach)
  • Vomiting (throwing up)
  • Abdominal pain
  • Diarrhea (may have blood)
  • Liver damage that can cause inflammation in the liver or jaundice (yellowing of the skin or eyes)
  • Damage to other organs

Steroids are the first line of treatment for acute GVHD. About a quarter of the patients that develop Acute GVHD may not respond to steroids and have steroid refractory GVHD (SR-aGVHD). Patients with SR-aGVHD may need other medications. SR-aGVHD, is a potentially life threatening condition. There is no standard treatment and it may not respond to treatment.

The goals of this study are to find out if combination drug therapy can treat SR-aGVHD.

Who can participate:

Patients between the ages of 6 months-75 years of age can participate.

 

Any patient with progressive Acute GVHD or Steroid Refractory GVHD can participate.

 

Patients with Refractory GVHD after Stem Cell or Bone Marrow Transplant can participate.

Patients may continue taking cyclosporine, tacrolimus, MMF, or sirolimus.

Patients should have an absolute neutrophil count (ANC) of greater than 500µL.

Patient should not be getting any other experimental therapy for aGvHD.

 

Patients with active uncontrolled life threatening infection (s) from viral, bacterial, fungal or other organisms will be excluded. Patients with HIV infection will be excluded.

Patients who are pregnant, breast feeding, or if sexually active and unwilling to use effective birth control for the duration of this study will be excluded.

Patients unable to give informed consent for the study will be excluded.

 

 

What will happen during the study:

Before you join the study, we will ask you about your medical history, and any medications you may be taking. It is important that you do not participate in the study if you are pregnant, breastfeeding or are likely to become pregnant during the study.

Your doctor will do a number of tests to determine if you are able to participate in this study. These tests include:

  • A physical exam that also measures the severity/stage of your SR-aGVHD.
  • Your ability to do regular daily activities (performance status).
  • ?Routine blood tests to check your blood counts, how well your liver and kidneys work, and levels of Acute GVHD prevention medications in your blood (if it applies to you).
  • Pregnancy test (if it applies to you).
  • We may also need a small piece (biopsy) of your skin, so we can study changes in your skin. If you agree to this, we will use numbing medicine before we take a small piece of skin with a special tool called a “punch”. If you have GVHD of your stomach/intestines/gut, then your transplant physician may consider getting a biopsy of the stomach/intestines/gut. If needed this will be most likely done while you are given a medication to sleep and will not feel any pain

If you decide to participate in this research study, you will receive the combination therapy once a week for four weeks. The medicines will be given through your broviac, port or through a vein in your arm. It will take about 4 or 5 hours to complete the 2-drug combination therapy each week. You will be given some medications first to help prevent any reactions to the study drugs.

  • You do not have to stop taking cyclosporine, tacrolimus, MMF or sirolimus if you are already on one of these medications.
  • Your absolute white blood cell count (ANC) needs to be above 500uL to participate
  • You will also be started on antibiotics, anti-fungal and antiviral medications to help prevent infections.

Once you finish the 4 weeks of study treatment, we will evaluate you weekly, then monthly, and as your condition improves the team will evaluate you less often. The study team will follow your health for up to 12 months after you first joined the study. During these visits, the team will evaluate your:

  • Physical examination
  • Acute GVHD stage
  • Ability to do daily activities (performance status)
  • Routine blood tests including your blood counts, how well your liver and kidneys work, and levels of Acute GVHD prevention medications in your blood (if it applies to you). Blood work to look for infections may be done if felt necessary.

Who to contact:

Jennifer Notestine
614-722-3637

Nationwide Children's Hospital
700 Children's Drive Columbus, Ohio 43205 614.722.2000