Plasma Cell Directed Therapy for Immune Thrombotic Thrombocytopenic Purpura (iTTP)

Joly, 2017, Thrombotic thrombocytopenic purpura, Blood, 129, 2836, 10.1182/blood-2016-10-709857 Kremer Hovinga, 2017, Thrombotic thrombocytopenic purpura, Nat Rev Dis Primers, 3, 17020, 10.1038/nrdp.2017.20 Alwan, 2019, Characterization and treatment of congenital thrombotic thrombocytopenic purpura, Blood, 133, 1644, 10.1182/blood-2018-11-884700 van Dorland, 2019, The International Hereditary Thrombotic Thrombocytopenic Purpura Registry: key findings at enrollment until 2017, Haematologica, 104, 2107, 10.3324/haematol.2019.216796 Hofmann, 2018, Targeting B cells and plasma cells in autoimmune diseases, Front Immunol, 9, 835, 10.3389/fimmu.2018.00835 Hiepe, 2016, Plasma cells as an innovative target in autoimmune disease with renal manifestations, Nat Rev Nephrol, 12, 232, 10.1038/nrneph.2016.20 Shin, 2022, Alterations in B- and circulating T-follicular helper cell subsets in immune thrombotic thrombocytopenic purpura, Blood Adv, 6, 3792, 10.1182/bloodadvances.2022007025 Mahévas, 2013, B cell depletion in immune thrombocytopenia reveals splenic long-lived plasma cells, J Clin Invest, 123, 432, 10.1172/JCI65689 Kojouri, 2004, Splenectomy for adult patients with idiopathic thrombocytopenic purpura: a systematic review to assess long-term platelet count responses, prediction of response, and surgical complications, Blood, 104, 2623, 10.1182/blood-2004-03-1168 Beloncle, 2012, Splenectomy and/or cyclophosphamide as salvage therapies in thrombotic thrombocytopenic purpura: the French TMA Reference Center experience, Transfusion, 52, 2436, 10.1111/j.1537-2995.2012.03578.x Rock, 1991, Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura, N Engl J Med, 325, 393, 10.1056/NEJM199108083250604 Benhamou, 2015, Cardiac troponin-I on diagnosis predicts early death and refractoriness in acquired thrombotic thrombocytopenic purpura. Experience of the French Thrombotic Microangiopathies Reference Center, J Thromb Haemost, 13, 293, 10.1111/jth.12790 Schaller, 2011, Anti-ADAMTS13 inhibitor boosting during plasma exchange therapy in acquired TTP Is the expression of a general dysregulation of the immune response, Blood, 118, 3299, 10.1182/blood.V118.21.3299.3299 Sun, 2019, Predictors of relapse and efficacy of rituximab in immune thrombotic thrombocytopenic purpura, Blood Adv, 3, 1512, 10.1182/bloodadvances.2019031039 Cuker, 2016, Adjuvant rituximab to prevent TTP relapse, Blood, 127, 2952, 10.1182/blood-2016-04-710475 Page, 2016, Rituximab reduces risk for relapse in patients with thrombotic thrombocytopenic purpura, Blood, 127, 3092, 10.1182/blood-2016-03-703827 Scully, 2011, A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura, Blood, 118, 1746, 10.1182/blood-2011-03-341131 Nguyen, 2008, Twice-daily plasma exchange for patients with refractory thrombotic thrombocytopenic purpura: the experience of the Oklahoma Registry, 1989 through 2006, Transfusion, 48, 349, 10.1111/j.1537-2995.2007.01530.x Balduini, 2010, High versus standard dose methylprednisolone in the acute phase of idiopathic thrombotic thrombocytopenic purpura: a randomized study, Ann Hematol, 89, 591, 10.1007/s00277-009-0877-5 Ferrara, 1999, Vincristine as salvage treatment for refractory thrombotic thrombocytopenic purpura, Ann Hematol, 78, 521, 10.1007/s002770050549 Ahmad, 2007, Mycophenolate mofetil in a case of relapsed, refractory thrombotic thrombocytopenic purpura, Eur J Haematol, 78, 449, 10.1111/j.1600-0609.2007.00832.x Cataland, 2007, An evaluation of ciclosporin and corticosteroids individually as adjuncts to plasma exchange in the treatment of thrombotic thrombocytopenic purpura, Br J Haematol, 136, 146, 10.1111/j.1365-2141.2006.06384.x Sayani, 2015, How I treat refractory thrombotic thrombocytopenic purpura, Blood, 125, 3860, 10.1182/blood-2014-11-551580 Coppo, 2015, Treatment of thrombotic thrombocytopenic purpura beyond therapeutic plasma exchange, Hematology Am Soc Hematol Educ Program, 2015, 637, 10.1182/asheducation-2015.1.637 Dane, 2018, Beyond plasma exchange: novel therapies for thrombotic thrombocytopenic purpura, Hematology Am Soc Hematol Educ Program, 2018, 539, 10.1182/asheducation-2018.1.539 Rottenstreich, 2016, The role of N-acetylcysteine in the treatment of thrombotic thrombocytopenic purpura, J Thromb Thrombolysis, 41, 678, 10.1007/s11239-015-1259-6 Scully, 2012, Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies, Br J Haematol, 158, 323, 10.1111/j.1365-2141.2012.09167.x Clark, 2015, A phase-II sequential case-series study of all patients presenting to four plasma exchange centres with presumed relapsed/refractory thrombotic thrombocytopenic purpura treated with rituximab, Br J Haematol, 170, 208, 10.1111/bjh.13408 Westwood, 2017, Rituximab prophylaxis to prevent thrombotic thrombocytopenic purpura relapse: outcome and evaluation of dosing regimens, Blood Adv, 1, 1159, 10.1182/bloodadvances.2017008268 McDonald, 2010, Rituximab pharmacokinetics during the management of acute idiopathic thrombotic thrombocytopenic purpura, J Thromb Haemost, 8, 1201, 10.1111/j.1538-7836.2010.03818.x Scully, 2019, Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura, N Engl J Med, 380, 335, 10.1056/NEJMoa1806311 Peyvandi, 2016, Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura, N Engl J Med, 374, 511, 10.1056/NEJMoa1505533 Khalesi, 2021, Bortezomib: a proteasome inhibitor for the treatment of autoimmune diseases, Inflammopharmacology, 29, 1291, 10.1007/s10787-021-00863-2 Pasquale, 2021, Bortezomib in autoimmune hemolytic anemia and beyond, Ther Adv Hematol, 12, 10.1177/20406207211046428 Moreau, 2011, Subcutaneous versus intravenous administration of bortezomib in patients with relapsed multiple myeloma: a randomised, phase 3, non-inferiority study, Lancet Oncol, 12, 431, 10.1016/S1470-2045(11)70081-X Neubert, 2008, The proteasome inhibitor bortezomib depletes plasma cells and protects mice with lupus-like disease from nephritis, Nat Med, 14, 748, 10.1038/nm1763 Alexander, 2015, The proteasome inhibitior bortezomib depletes plasma cells and ameliorates clinical manifestations of refractory systemic lupus erythematosus, Ann Rheum Dis, 74, 1474, 10.1136/annrheumdis-2014-206016 Schneider-Gold, 2017, Bortezomib in severe MuSK-antibody positive myasthenia gravis: first clinical experience, Ther Adv Neurol Disord, 10, 339, 10.1177/1756285617721093 Hartono, 2014, Bortezomib therapy for nephrotic syndrome due to idiopathic membranous nephropathy, J Nephrol, 27, 103, 10.1007/s40620-013-0028-x Ratnasingam, 2016, Bortezomib-based antibody depletion for refractory autoimmune hematological diseases, Blood Adv, 1, 31, 10.1182/bloodadvances.2016001412 Trivedi, 2009, Abrogation of anti-HLA antibodies via proteasome inhibition, Transplantation, 87, 1555, 10.1097/TP.0b013e3181a4b91b Sorvillo, 2012, The macrophage mannose receptor promotes uptake of ADAMTS13 by dendritic cells, Blood, 119, 3828, 10.1182/blood-2011-09-377754 Park, 2013, Antibody depletion by bortezomib through blocking of antigen presentation, N Engl J Med, 368, 1364, 10.1056/NEJMc1301264 Berges, 2008, Proteasome inhibition suppresses essential immune functions of human CD4+ T cells, Immunology, 124, 234, 10.1111/j.1365-2567.2007.02761.x Lin, 2004, Flow cytometric immunophenotypic analysis of 306 cases of multiple myeloma, Am J Clin Pathol, 121, 482, 10.1309/74R4TB90BUWH27JX Halliley, 2015, Long-lived plasma cells are contained within the CD19(-)CD38(hi)CD138(+) subset in human bone marrow, Immunity, 43, 132, 10.1016/j.immuni.2015.06.016 Malavasi, 2008, Evolution and function of the ADP ribosyl Cyclase/CD38 gene family in physiology and pathology, Physiol Rev, 88, 841, 10.1152/physrev.00035.2007 De Weers, 2011, Daratumumab, a novel therapeutic human CD38 monoclonal antibody, induces killing of multiple myeloma and other hematological tumors, J Immunol, 186, 1840, 10.4049/jimmunol.1003032 Overdijk, 2015, Antibody-mediated phagocytosis contributes to the anti-tumor activity of the therapeutic antibody daratumumab in lymphoma and multiple myeloma, MAbs, 7, 311, 10.1080/19420862.2015.1007813 Marco Jansen, 2012, Daratumumab, a human CD38 antibody induces apoptosis of myeloma tumor cells via Fc receptor-mediated crosslinking, Blood, 120, 2974, 10.1182/blood.V120.21.2974.2974 Ostendorf, 2020, Targeting CD38 with daratumumab in refractory systemic lupus erythematosus, N Engl J Med, 383, 1149, 10.1056/NEJMoa2023325 Crickx, 2021, Daratumumab, an original approach for treating multi-refractory autoimmune cytopenia, Haematologica, 106, 3198, 10.3324/haematol.2021.279232 Jain, 2021, Daratumumab for refractory warm autoimmune hemolytic anemia, Ann Hematol, 100, 1351, 10.1007/s00277-020-04063-w Shortt, 2013, ADAMTS13 Antibody Depletion by Bortezomib in Thrombotic Thrombocytopenic Purpura, N Engl J Med, 368, 90, 10.1056/NEJMc1213206 Cuker, 2021, Redefining outcomes in immune TTP: an international working group consensus report, Blood, 137, 1855, 10.1182/blood.2020009150 Patel, 2016, Rituximab-refractory thrombotic thrombocytopenic purpura responsive to intravenous but not subcutaneous bortezomib, Transfusion, 56, 970, 10.1111/trf.13465 van den Berg, 2022, Daratumumab for immune thrombotic thrombocytopenic purpura, Blood Adv, 6, 993, 10.1182/bloodadvances.2021005124 Mazepa, 2014, Bortezomib induces clinical remission and reduction of ADAMTS13 inhibitory antibodies in relapsed refractory idiopathic thrombotic thrombocytopenic purpura, Br J Haematol, 164, 900, 10.1111/bjh.12696 Maschan, 2021, Bortezomib induces long-term remission in children with immune thrombotic thrombocytopenic purpura, refractory to plasma exchange, glucocorticoids, and rituximab: A report on two cases, Pediatr Blood Cancer, 68, e28818, 10.1002/pbc.28818 Zheng, 2020, ISTH guidelines for treatment of thrombotic thrombocytopenic purpura, J Thromb Haemost, 18, 2496, 10.1111/jth.15010 Fox, 2018, Consensus opinion on diagnosis and management of thrombotic microangiopathy in Australia and New Zealand, Intern. Med. J, 48, 624, 10.1111/imj.13804 George, 2013, The Oklahoma thrombotic thrombocytopenic purpura-haemolytic uraemic syndrome registry. a model for clinical research, education and patient care, Hamostaseologie, 33, 105, 10.5482/HAMO-12-10-0016 2022 Richardson, 2009, Single-agent bortezomib in previously untreated multiple myeloma: efficacy, characterization of peripheral neuropathy, and molecular correlations with response and neuropathy, J Clin Oncol, 27, 3518, 10.1200/JCO.2008.18.3087 Sahin, 2019, Refractory thrombotic thrombocytopenic purpura in a patient with Kaposi sarcoma, Transfus Apher Sci, 58, 187, 10.1016/j.transci.2019.02.003 Lancelot, 2021, Refractory thrombotic thrombocytopenic purpura related to checkpoint inhibitor immunotherapy, Transfusion, 61, 322, 10.1111/trf.16117 Xiao, 2014, Incidence and risk of cardiotoxicity associated with bortezomib in the treatment of cancer: a systematic review and meta-analysis, PLoS One, 9, e87671, 10.1371/journal.pone.0087671 Chen, 2017, Cardiac events during treatment with proteasome inhibitor therapy for multiple myeloma, Cardio-Oncology, 3, 4, 10.1186/s40959-017-0023-9 Westwood, 2013, Rituximab for thrombotic thrombocytopenic purpura: benefit of early administration during acute episodes and use of prophylaxis to prevent relapse, J Thromb Haemost, 11, 481, 10.1111/jth.12114 Ichikawa, 2012, Beneficial effect of novel proteasome inhibitors in murine lupus via dual inhibition of type I interferon and autoantibody-secreting cells, Arthritis Rheum, 64, 493, 10.1002/art.33333 Seavey, 2012, Novel, orally active, proteasome inhibitor, delanzomib (CEP-18770), ameliorates disease symptoms and glomerulonephritis in two preclinical mouse models of SLE, Int Immunopharmacol, 12, 257, 10.1016/j.intimp.2011.11.019 Lodhi, 2015, Thrombotic microangiopathy associated with proteasome inhibitors, Clin Kidney J, 8, 632, 10.1093/ckj/sfv059 Atallah-Yunes, 2018, Drug-induced thrombotic microangiopathy due to cumulative toxicity of ixazomib, Case Rep Hematol, 2018 Jagannath, 2018, Elotuzumab monotherapy in patients with smouldering multiple myeloma: a phase 2 study, Br J Haematol, 182, 495, 10.1111/bjh.15384 Zonder, 2012, A phase 1, multicenter, open-label, dose escalation study of elotuzumab in patients with advanced multiple myeloma, Blood, 120, 552, 10.1182/blood-2011-06-360552 Liang, 2022, Response to belimumab in thrombotic thrombocytopenic purpura associated with systemic lupus erythematosus: a case-based review, Clin Rheumatol, 41, 2561, 10.1007/s10067-022-06155-6 van Balen, 2014, Refractory thrombotic thrombocytopenic purpura in a 16-year-old girl: successful treatment with bortezomib, Eur J Haematol, 92, 80, 10.1111/ejh.12206 Yates, 2014, Bortezomib for chronic relapsing thrombotic thrombocytopenic purpura: a case report, Transfusion, 54, 2064, 10.1111/trf.12614 Acedillo, 2016, Treatment of severe, refractory and rapidly evolving thrombotic thrombocytopenic purpura, BMJ Case Rep, 2016 Patriquin, 2016, Bortezomib in the treatment of refractory thrombotic thrombocytopenic purpura, Br J Haematol, 173, 779, 10.1111/bjh.13993 Pandey, 2017, Remission of severe, relapsed, and refractory TTP after multiple cycles of bortezomib, Case Rep Hematol, 2017, 9681832 Sun, 2018, Relapsed/refractory acquired thrombotic thrombocytopenic purpura in a patient with Sjögren syndrome: Case report and review of the literature, Medicine (Baltimore), 97, e12989, 10.1097/MD.0000000000012989 Hernández Lorente, 2019, Effectiveness of bortezomib in the treatment of thrombotic thrombocytopenic purpura: Case report, Med Clin (Barc), 152, 244, 10.1016/j.medcli.2018.06.002 Baseri, 2019, Survival of a Jehovah’s Witness with thrombotic thrombocytopenic purpura without using plasma: A case report and review of the literature, J Clin Apher, 34, 623, 10.1002/jca.21710 Bavli, 2021, Complete ADAMTS13 remission in a patient with refractory autoimmune-mediated thrombotic thrombocytopenic purpura after infliximab, Transfus Apher Sci, 60, 10.1016/j.transci.2021.103213 Azapagasi, 2021, Successful treatment with bortezomib for refractory and complicated acquired thrombotic thrombocytopenic purpura in an adolescent girl, J Pediatr Hematol Oncol, 43, e587, 10.1097/MPH.0000000000002026