Table 1.

Adverse reactions of commonly used therapies in refractory chronic GVHD14

AgentPotential major adverse effects (with major study citations)Common (>10%) generally less severe adverse effects
BortezomibPeripheral neuropathy, thrombocytopenia, malignancy relapse106Herpes virus reactivation
ECPVascular access complications107Thrombocytopenia
FAMNew FDA MedWatch warning; warning only applies to azithromycin use in prophylactic (not treatment) setting108,109
Ibrutinib (Imbruvica R)Pneumonia,29 impaired platelet functionFatigue, muscle pain, peripheral edema
ImatinibPeripheral edema
Interleukin-2Injection site induration, infections36Constitutional flu-like symptoms
MMF (Cellcept)Viral reactivation, hypertension, pneumonia, posttransplantation lymphoproliferative disease110GI toxicity, neutropenia, leukopenia
PamolidomideTremor, muscle cramps, peripheral neuropathy111Skin rash
Rituximab (Rituxan R)Infection, late neutropenia38,39,112B lymphopenia
Ruxolitinib (Jakafi R)Viral reactivation/infection, bacterial infections35Cytopenias
Sirolimus (Rapamune)TAM when used in combination with calcineurin inhibitors, renal insufficiency,113 proteinuriaPeripheral edema, hyperlipidemia, cytopenias
  • This list of agents represents a fraction of agents being actively evaluated. Preferred use of any agent still requires validation via larger clinical trials.

  • ECP, extracorporeal photopheresis; FAM, fluticasone, azithromycin, and montelukast; FDA, US Food and Drug Administration; GI, gastrointestinal; MMF, mycophenolate mofetil; TAM, transplantation-associated microangiopathy.