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GRANADA, SPAIN—A monoclonal antibody can resolve co-existing hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) and graft-versus-host disease (GVHD), according to a case study.
The antibody is OMS721, and it targets MASP-2, a pro-inflammatory protein target involved in activation of the complement system.
The case of OMS721 ameliorating HSCT-TMA and GVHD was presented at the European Society for Blood and Marrow Transplantation Crash Course on Diagnosis and Treatment of Noninfectious Complications after HCT.
The patient was a participant in an ongoing phase 2 trial of thrombotic microangiopathies, including HSCT-TMA. The trial is sponsored by Omeros Corporation, the company developing OMS721.
The patient was an adult male with post-transplant TMA persisting at least 2 weeks following calcineurin inhibitor modification.
The patient had undergone HSCT for T-cell acute lymphoblastic leukemia. His post-transplant course was complicated by multiple episodes of steroid-refractory grade 4 GVHD, cytomegalovirus infection, and HSCT-TMA.
After 2 prior episodes of GVHD, the patient presented with bloody diarrhea. Intestinal biopsy demonstrated both HSCT-TMA and GVHD. No infections were identified.
The patient also had new-onset neurological symptoms of paresthesias, tetraparesis, and a neurogenic bladder, which have been reported as neurological manifestations of GVHD and TMA.
The patient was unable to walk due to the tetraparesis and required blood transfusions at least once daily. Hematological markers demonstrated HSCT-TMA with thrombocytopenia, elevated lactate dehydrogenase, and schistocytes.
Two weeks prior to starting OMS721, the patient’s immunosuppression (cyclosporine) had been decreased, and, given his history of steroid-refractory GVHD, he was receiving only low-dose corticosteroids. He received no other GVHD treatment.
After 2 OMS721 doses, the patient’s bloody diarrhea resolved, and his hematological markers improved. After 4 OMS721 doses, he was able to walk with help.
The patient completed 8 weeks of OMS721 treatment and has been doing well at home. All signs and symptoms of HSCT-TMA and all clinical symptoms of GVHD have resolved. His neurological symptoms have continued to improve.
“This patient’s marked response to OMS721 treatment was very gratifying,” said Anna Grassi, MD, of Azienda Ospedaliera Papa Giovanni XXIII in Bergamo, Italy.
“The cause of his neurological symptoms is not clear but may be a manifestation of GVHD or other endothelial injury. Prior to OMS721 treatment, this patient was deteriorating and at high risk for early death. The improvement of GVHD, H[S]CT-TMA, and the neurological symptoms following OMS721 treatment is promising.” 
GRANADA, SPAIN—A monoclonal antibody can resolve co-existing hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) and graft-versus-host disease (GVHD), according to a case study.
The antibody is OMS721, and it targets MASP-2, a pro-inflammatory protein target involved in activation of the complement system.
The case of OMS721 ameliorating HSCT-TMA and GVHD was presented at the European Society for Blood and Marrow Transplantation Crash Course on Diagnosis and Treatment of Noninfectious Complications after HCT.
The patient was a participant in an ongoing phase 2 trial of thrombotic microangiopathies, including HSCT-TMA. The trial is sponsored by Omeros Corporation, the company developing OMS721.
The patient was an adult male with post-transplant TMA persisting at least 2 weeks following calcineurin inhibitor modification.
The patient had undergone HSCT for T-cell acute lymphoblastic leukemia. His post-transplant course was complicated by multiple episodes of steroid-refractory grade 4 GVHD, cytomegalovirus infection, and HSCT-TMA.
After 2 prior episodes of GVHD, the patient presented with bloody diarrhea. Intestinal biopsy demonstrated both HSCT-TMA and GVHD. No infections were identified.
The patient also had new-onset neurological symptoms of paresthesias, tetraparesis, and a neurogenic bladder, which have been reported as neurological manifestations of GVHD and TMA.
The patient was unable to walk due to the tetraparesis and required blood transfusions at least once daily. Hematological markers demonstrated HSCT-TMA with thrombocytopenia, elevated lactate dehydrogenase, and schistocytes.
Two weeks prior to starting OMS721, the patient’s immunosuppression (cyclosporine) had been decreased, and, given his history of steroid-refractory GVHD, he was receiving only low-dose corticosteroids. He received no other GVHD treatment.
After 2 OMS721 doses, the patient’s bloody diarrhea resolved, and his hematological markers improved. After 4 OMS721 doses, he was able to walk with help.
The patient completed 8 weeks of OMS721 treatment and has been doing well at home. All signs and symptoms of HSCT-TMA and all clinical symptoms of GVHD have resolved. His neurological symptoms have continued to improve.
“This patient’s marked response to OMS721 treatment was very gratifying,” said Anna Grassi, MD, of Azienda Ospedaliera Papa Giovanni XXIII in Bergamo, Italy.
“The cause of his neurological symptoms is not clear but may be a manifestation of GVHD or other endothelial injury. Prior to OMS721 treatment, this patient was deteriorating and at high risk for early death. The improvement of GVHD, H[S]CT-TMA, and the neurological symptoms following OMS721 treatment is promising.”
GRANADA, SPAIN—A monoclonal antibody can resolve co-existing hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) and graft-versus-host disease (GVHD), according to a case study.
The antibody is OMS721, and it targets MASP-2, a pro-inflammatory protein target involved in activation of the complement system.
The case of OMS721 ameliorating HSCT-TMA and GVHD was presented at the European Society for Blood and Marrow Transplantation Crash Course on Diagnosis and Treatment of Noninfectious Complications after HCT.
The patient was a participant in an ongoing phase 2 trial of thrombotic microangiopathies, including HSCT-TMA. The trial is sponsored by Omeros Corporation, the company developing OMS721.
The patient was an adult male with post-transplant TMA persisting at least 2 weeks following calcineurin inhibitor modification.
The patient had undergone HSCT for T-cell acute lymphoblastic leukemia. His post-transplant course was complicated by multiple episodes of steroid-refractory grade 4 GVHD, cytomegalovirus infection, and HSCT-TMA.
After 2 prior episodes of GVHD, the patient presented with bloody diarrhea. Intestinal biopsy demonstrated both HSCT-TMA and GVHD. No infections were identified.
The patient also had new-onset neurological symptoms of paresthesias, tetraparesis, and a neurogenic bladder, which have been reported as neurological manifestations of GVHD and TMA.
The patient was unable to walk due to the tetraparesis and required blood transfusions at least once daily. Hematological markers demonstrated HSCT-TMA with thrombocytopenia, elevated lactate dehydrogenase, and schistocytes.
Two weeks prior to starting OMS721, the patient’s immunosuppression (cyclosporine) had been decreased, and, given his history of steroid-refractory GVHD, he was receiving only low-dose corticosteroids. He received no other GVHD treatment.
After 2 OMS721 doses, the patient’s bloody diarrhea resolved, and his hematological markers improved. After 4 OMS721 doses, he was able to walk with help.
The patient completed 8 weeks of OMS721 treatment and has been doing well at home. All signs and symptoms of HSCT-TMA and all clinical symptoms of GVHD have resolved. His neurological symptoms have continued to improve.
“This patient’s marked response to OMS721 treatment was very gratifying,” said Anna Grassi, MD, of Azienda Ospedaliera Papa Giovanni XXIII in Bergamo, Italy.
“The cause of his neurological symptoms is not clear but may be a manifestation of GVHD or other endothelial injury. Prior to OMS721 treatment, this patient was deteriorating and at high risk for early death. The improvement of GVHD, H[S]CT-TMA, and the neurological symptoms following OMS721 treatment is promising.”