Disease Description (Causes and Symptoms)
- Name: Graft-versus-host disease (GVHD) — the commonly used medical term.
- Causes and pathophysiology: After allogeneic hematopoietic stem cell transplantation or blood product transplantation, mature donor T lymphocytes (or other immune cells) recognize antigens in host tissues (HLA or minor histocompatibility antigens) as foreign and immunologically attack host organs, including the skin, liver, and gastrointestinal tract. It is classified as acute (usually within 100 days after transplantation) or chronic (>100 days, including fibrosis and autoimmune-like features).
- Main symptoms:
- Skin: Erythematous rash (often involving the auricles and palms/soles); in severe cases, blisters and epidermal detachment (which may cause shock if extensive).
- Liver: Jaundice and elevated liver function tests (increased bilirubin and AST/ALT).
- Gastrointestinal tract: Nausea, vomiting, decreased appetite, abdominal pain, and frequent watery diarrhea (electrolyte loss and weight loss).
- Systemic: Fever, increased opportunistic infections due to impaired immune function, and cytopenia.
- Chronic GVHD can cause chronic disability, including scleroderma-like skin changes, dry mouth and eyes, chronic pulmonary lesions (bronchiolitis), and chronic liver and gastrointestinal damage.
Treatment
- First-line treatment for acute GVHD: High-dose systemic steroids (e.g., methylprednisolone/prednisone).
- Steroid-refractory or adjunctive treatment:
- Immunosuppressants/anticancer agents: Cyclosporine and tacrolimus (for prevention and treatment), mycophenolate, sirolimus, antithymocyte globulin (ATG), etc.
- Targeted therapies/new drugs: Ruxolitinib (a JAK inhibitor) — used as an option for steroid-refractory acute/chronic GVHD. Ibrutinib has been approved for chronic GVHD in some cases.
- Other: Extracorporeal photopheresis (photopheresis, ECP) is useful in chronic or steroid-refractory cases. Monoclonal antibodies (e.g., alemtuzumab) may be considered in severe cases.
- Supportive care: Fluids and electrolyte replacement, nutritional support (TPN if oral intake is not possible), pain and nausea management, and skin, oral, and eye care.
- Infection prevention and management: Prevent CMV and HSV reactivation (periodic PCR monitoring and antivirals), prevent PCP (TMP-SMX), and use antifungal and antiviral prophylaxis when necessary. For vaccinations, establish a revaccination plan according to the timing after transplantation.
- Prevention strategies: Maximize HLA matching, T-cell modulation before/during transplantation (partial T-cell depletion), post-transplant immunosuppressants (e.g., cyclosporine + methotrexate), and, more recently, post-transplant low-dose cyclophosphamide (PTCy), which is effective in preventing acute GVHD.
- Precautions (during and after treatment): The risk of infection is increased (immediate evaluation is needed in case of fever, respiratory symptoms, or a localized infection); long-term steroid adverse effects (osteoporosis, hyperglycemia, gastric ulcers, infection, etc.) require monitoring and prevention.
Checklist Before an Outpatient Visit (Including Example Questions)
Materials and test records to prepare:
- Transplant-related information: Transplantation date, relationship to the donor, HLA matching status, type of hematopoietic stem cells (bone marrow/peripheral blood/cord blood), conditioning regimen used, and GVHD prophylaxis medications.
- Current medications (immunosuppressants, antivirals, antifungals, steroids, etc.), including doses and start dates.
- Recent test results: CBC, liver function tests (bilirubin and AST/ALT), electrolytes, kidney function, CMV/EBV PCR, stool tests (bleeding and intestinal infection), and results of any recent biopsies (skin, liver, or intestine) and pathology reports.
- Symptom records: Photos of the rash (with the date taken indicated), frequency and amount of diarrhea (e.g., episodes/day and whether watery), the onset date and progression of jaundice, weight changes, presence of fever, shortness of breath, etc.
- History of previous infections and vaccination records, allergies, and other underlying conditions (diabetes, osteoporosis, etc.).
Questions to prepare for the outpatient visit (it is useful for patients to record these themselves):
- When did the rash begin, and on which body area did it start? Can I provide photos?
- How many episodes of diarrhea occur per day, and what is the average amount? Is there blood or mucus?
- What has been the recent change in weight (over the past 1–2 weeks)?
- What medications am I currently taking, what are the exact dosing times, and have there been any recent changes?
Examples of General Questions to Ask the Doctor
- How is the severity (grade) of my current condition assessed? What is the expected prognosis?
- What are the recommended first-line and alternative treatments (including adverse effects and expected benefits)?
- What is the plan for stopping or tapering my current immunosuppressants and steroids?
- Which medications should I take to prevent infections, and for how long? What are the adverse effects?
- What precautions should I take in daily life (meals, going out, vaccinations, returning to work, etc.)?
Examples of Disease-Specific Questions
- If the skin rash spreads or epidermal detachment begins, when would you recommend hospitalization or a skin biopsy?
- If the amount of diarrhea is OO mL per day (e.g., >1,000 mL), should intravenous fluids/TPN or inpatient treatment be considered? Also, what is the first-line medication-based response to the diarrhea?