- |||||||||| Decadron (dexamethasone) / Merck (MSD), Gamifant (emapalumab) / SOBI, Soliris (eculizumab) / Alexion Pharma
THE USE OF EMAPALUMAB AS TREATMENT FOR A CRITICALLY ILL PATIENT WITH FAMILIAL HLH () - Apr 3, 2020 - Abstract #ASPHO2020ASPHO_382;
In our patient with life threatening complications due to shiga toxin induced HUS, Emapalumab treated his HLH to allow recovery of his organ function. Given our experience, this medication appears to be an excellent option for the treatment of HLH, even in patients with significant co-morbidities.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, doxorubicin hydrochloride / Generic mfg., cyclophosphamide intravenous / Generic mfg.
GENE NEGATIVE ATYPICAL HUS IN A PATIENT WITH METASTATIC WILMS' TUMOR () - Apr 3, 2020 - Abstract #ASPHO2020ASPHO_155;
Eculizumab use allowed her to complete a modified chemotherapy regimen with improvement in her aHUS and continued tumor remission. There have been reports of successful treatment of drug induced aHUS in adults, however nothing has been reported in children.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
EVOLUTION OF MODERATE APLASTIC ANEMIA INTO SUBCLINICAL PNH/AA & SUCCESSFUL TREATMENT WITH HAPLO- BMT () - Apr 3, 2020 - Abstract #ASPHO2020ASPHO_135;
Since our patient was starting college, we treated her with Immunosuppressive Therapy (IST) consisting of equine Anti-thymocyte globulin & Cyclosporine...Our patient’s LDH was normal; unlikely to respond to eculizumab... The case illustrates diagnostic challenges of patients presenting with pancytopenia; the overlap and possible evolution of disease classifications; concepts like HDA in PNH to help guide decision making; new diagnostic testing; the successful use of RIC and haplo BMT in a patient with PNH/AA and a normocellular marrow.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
EVANS SYNDROME AND THROMBOTIC MICROANGIOPATHY OCCURRING SIMULTANEOUSLY IN ONE PATIENT () - Apr 3, 2020 - Abstract #ASPHO2020ASPHO_134;
Hypertension is an expected side effect of prolonged steroid exposure, and if severe can lead to renal damage including TMA. Evans syndrome has not been previously associated with TMA, although Coombs positivity has been rarely described in patients with post-pneumococcal HUS and thrombotic thrombocytopenic purpura.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo, Rituxan Hycela (rituximab/hyaluronidase) / Biogen, Roche, Halozyme
Review, Journal: Immunosuppressive and Immunomodulatory Therapies for Neuromuscular Diseases Part II: New and Novel Agents. (Pubmed Central) - Mar 31, 2020
This second article focuses on new and novel immune-based therapies including: eculizumab, a complement inhibitor approved for acetylcholine receptor antibody-positive myasthenia gravis (MG); rituximab, a B-cell depletion therapy with evolving indications in neuromuscular diseases; and the subcutaneous formulation of immunoglobulin G (SCIG) that gained approval for use in chronic inflammatory demyelinating polyneuropathy (CIDP) in 2018. Finally, several novel antigen-specific drugs at different stages of investigation in neuromuscular disease are also reviewed.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
RECURRENT THROMBOTIC MICROANGIOPATHY AND LONG-TERM OUTCOMES () - Mar 27, 2020 - Abstract #NKFCSM2020NKF_CSM_371;
Among those who did not receive treatment with Eculizumab, 15 (30%) with recurrent TMA and 35 (70%) patients with de novo TMA progressed to ESKD (p=0.157)... In this cohort with long-term follow-up, recurrent TMA did not pose significantly higher odds of ESKD or mortality compared to those with de novo TMA, but these findings need to be validated in larger cohorts.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, cyclophosphamide intravenous / Generic mfg.
Unusual renal recovery after severe Lupus Nephritis (LN) complicated by Thrombotic Microangiopathy (TMA) () - Mar 27, 2020 - Abstract #NKFCSM2020NKF_CSM_343;
23 years old African American woman with LN class IV at the age of 16 treated successfully with mycophenolate mofetil (MMF) and steroids at that time...She continued to receive HD three times per week, completed 6 months of therapy with Cyclophosphamide and Eculizumab, transitioned to MMF/steroids for maintenance... Late recovery after severe TMA complicating LN is possible, and expectant management is warranted.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Pregnancy-associated complement-mediated aHUS () - Mar 27, 2020 - Abstract #NKFCSM2020NKF_CSM_342;
In contrast to ADAMTS13 deficiency-related TMA, complement-mediated aHUS is more often seen in the postpartum period. Higher rates of infection and bleeding around time of delivery are thought to potentiate complement pathway activation.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Cytomegalovirus Induced Thrombotic Microangiopathy of Kidney Allograft () - Mar 27, 2020 - Abstract #NKFCSM2020NKF_CSM_177;
In conclusion, while rejection is the most common cause of post-transplant TMA, due consideration must be given to infection as a factor in its pathogenesis. Consequently, management decisions that take the underlying cause of TMA into account are much more likely to be more effective in these cases.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
An Eculizumab-Resistant TMA? () - Mar 27, 2020 - Abstract #NKFCSM2020NKF_CSM_111;
In retrospective trials, treatment of complement-mediated TMA with eculizumab achieved complete response in around two-thirds of patients. Individuals not responding to treatment with eculizumab may have rare genetic variants in non-complement genes or polymorphism in the C5 gene.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Avastin (bevacizumab) / Roche
Eculizumab for Bevacizumab mediated TMA () - Mar 27, 2020 - Abstract #NKFCSM2020NKF_CSM_50;
Individuals not responding to treatment with eculizumab may have rare genetic variants in non-complement genes or polymorphism in the C5 gene. Anticompliment Therapy might be an alternative to plasma exchange for drug induced TMA.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Drug-induced TMA from trimethoprim-sulfamethoxazole () - Mar 27, 2020 - Abstract #NKFCSM2020NKF_CSM_23;
Trimethoprim-sulfamethoxazole is a rare cause of thrombotic microangiopathy, and the exact mechanism is not understood. Our case highlights the importance of considering TMP-SMX as a potential cause in patients presenting with TMA.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Journal: Eculizumab Is Safe and Effective as a Long-term Treatment for Protein-losing Enteropathy Due to CD55 Deficiency. (Pubmed Central) - Mar 26, 2020
Targeted therapy with the terminal complement inhibitor eculizumab has positive clinical and laboratory outcomes in PLE related to CD55 loss-of-function mutations, previously a life-threatening condition. Our results demonstrate the potential of genetic diagnosis to guide tailored treatment, and underscore the significant role of the complement system in the intestine.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
Review, Journal: Immunosuppressive and Immunomodulatory Therapies for Neuromuscular Diseases Part I: Traditional Agents. (Pubmed Central) - Mar 24, 2020
Part I provides an update on the evidence and use of traditional therapies, such as corticosteroids, PLEX, intravenously delivered IgG (IVIG), and steroid-sparing immunosuppressive drugs. Part II focuses on the recently FDA-approved therapies eculizumab and subcutaneous IgG (SCIG), the current indications for rituximab in neuromuscular disease, and on novel immunosuppressive therapeutic approaches underdevelopment.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Clinical, Journal: C5 Convertase Blockade in Membranoproliferative Glomerulonephritis: A Single-Arm Clinical Trial. (Pubmed Central) - Mar 20, 2020
P4
However, it is time to consider conducting a long-term study to learn about the safety and efficacy of this approach, which may have a major financial advantage for patients. Eculizumab blunted terminal complement activation in all patients with immune complex-mediated MPGN or C3 glomerulonephritis and nephrotic syndrome, but persistently reduced proteinuria in just a subgroup.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
Multifocal Motor Neuropathy with Respiratory Muscle Involvement: A Case Report (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area H) - Mar 15, 2020 - Abstract #ATS2020ATS_9322;
Rituxan has now been added to his medical management to further optimize his respiratory status since his improvement with IVIg plateaued. This case report elucidates the similarities between ALS and MMN, the need for earlier, accurate diagnosis and treatment, and the management of severe, progressive, IVIg resistant MMN.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
Diffuse Alveolar Hemorrhage in Primary Antiphospholipid Syndrome (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area H) - Mar 15, 2020 - Abstract #ATS2020ATS_9191;
This presents a challenge in the management of pulmonary hemostasis both in the acute phase and maintenance of remission.Case PresentationA 55 year-old gentleman with a 10-year history of primary APS with recurrent deep vein thromboses on rivaroxaban, and proteinuric chronic kidney disease stage III, who presented with acute-on-chronic progressive hypoxic respiratory failure and acute tubular necrosis...Efforts to transition to steroid-sparing interventions included a brief course of rituximab, hydroxychloroquine and Intravenous Immune Globulin...Eculizumab, an anti-C5 antibody, is being used as a salvage therapy for renal manifestations of APS-associated TMA. Additional investigation is required to further elucidate the mechanism of eculizumab in the lungs and its role in treating pulmonary manifestations of APS.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Enbrel (etanercept) / Pfizer, Amgen, cyclophosphamide intravenous / Generic mfg.
Pulmonary Veno-Occlusive Disease- An Underrecognized Cause of Pulmonary Hypertension Following Pediatric Hematopoietic Stem Cell Transplantation (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area H) - Mar 15, 2020 - Abstract #ATS2020ATS_5676;
We present a case of a 10-year-old male who underwent a matched sibling hematopoietic stem cell transplant (HSCT) for idiopathic aplastic anemia following a fludarabine/cyclophosphamide/ATG myeloablative regimen and subsequently developed respiratory failure from biopsy-proven pulmonary veno-occlusive disease (PVOD)...The patient was given a presumed diagnosis of idiopathic pulmonary syndrome (IPS) and started on etanercept and methylprednisolone...Pulse-dosed methylprednisolone was given for a presumed IPS flare, and eculizumab was continued for his renal disease...An association between TMA and pulmonary arterial disease has been previously reported, but this case highlights that pulmonary venous disease can also occur in HSCT patients with TMA. Early recognition of PVOD, along with the potential association of pulmonary vascular disease with TMA, may be of benefit in managing these devastating HSCT complications.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Could It Be Prostate Cancer? A Rare Case of Eculizumab Use in Cancer-Associated Thrombotic Microangiopathy (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area G) - Mar 15, 2020 - Abstract #ATS2020ATS_5466;
Eculizumab was FDA-approved for use in TMA patients with normal ADAMTS13 activity who did not respond to therapeutic plasma exchange as would be expected in TTP, though it has not been adequately investigated in individuals with known malignancy. This case exhibits one of the first documented examples of efficacious eculizumab use in cancer-associated TMA.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, doxycycline / Generic mfg., Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
Catastrophic Antiphospholipid Syndrome (CAPS) Triggered by Pneumonia and Successfully Treated with Rituximab (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area A) - Mar 15, 2020 - Abstract #ATS2020ATS_5050;
The goal of treatment in CAPS is to treat the underlying trigger, such as infection, and to modulate the immune response to the circulating antiphospholipid antibodies that are attacking the endothelial cells and activating the complement system. Because of the role of the immune system in the pathogenesis of the syndrome, the treatment mainstays of glucocorticoids, anticoagulants, and plasma exchange can be augmented with specific immune modulators such as eculizumab and rituximab, as was used in this case.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Spitting Blood and Casts with Mud: A Rare and Unusual Presentation of C3 Glomerulonephritis (PENNSYLVANIA CONVENTION CENTER, Hall D-E (200 Level), Area A) - Mar 15, 2020 - Abstract #ATS2020ATS_5038;
This patient had PRS secondary to C3GN; even more unusual, the initial presentation of C3GN was hemoptysis. Most often, the treatment modalities will involve pulse dose steroids, plasmapheresis, and immunosuppressive agents (mycophenolate mofetil or eculizumab).
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Rituxan (rituximab) / Roche, Biogen, Zenyaku Kogyo
Lung Transplantation and Donor-Specific Antigens: Clinical and Serologic Factors Associated with Survival and Allograft Dysfunction (PENNSYLVANIA CONVENTION CENTER, Hall G (100 Level)) - Mar 15, 2020 - Abstract #ATS2020ATS_3052;
Treatment with bortezomib, thymoglobulin, and eculizumab were reserved for those with concomitant rejection or failure of initial treatment to clear DSA and were not associated with improved OS or BOS-free survival.ConclusionPatients who developed DSA but had clearance had survival curves similar to those who never developed DSA, while persistent DSA presence showed worse survival. Lower magnitude of DSA at onset and peak is associated with spontaneous DSA clearance, and treatment with PLEX or rituximab is associated with DSA clearance.
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