- |||||||||| Actemra IV (tocilizumab) / Roche, JW Pharma
[VIRTUAL] CARDIAC TAMPONADE AS A PRESENTING FEATURE OF METASTATIC GI CARCINOMA () - Oct 13, 2021 - Abstract #CHEST2021CHEST_419;
She was later diagnosed with aplastic anemia and underwent a conditioning regimen prior to peripheral blood stem cell transplantation (PBSCT) with cyclophosphamide, fludarabine, total body irradiation, and rabbit anti-thymocyte globulin (ATG). Despite trying rituximab, dexamethasone, etoposide, tocilizumab and Emapalumab, our transplant patient exhibits a unique and ambiguous intersection of EBV-associated refractory HLH, unresponsive to treatments.
- |||||||||| Soliris (eculizumab) / AstraZeneca
Clinical, Journal: Thinking Beyond HLH: Clinical Features of Patients with Concurrent Presentation of Hemophagocytic Lymphohistiocytosis and Thrombotic Microangiopathy. (Pubmed Central) - Sep 19, 2021
Our observations suggest co-activation of both interferon and complement pathways as a potential culprit in the evolution of thrombotic microangiopathy in patients with inflammatory disorders like refractory HLH and may offer novel therapeutic approaches for these critically ill patients. TMA should be considered in children with HLH and multi-organ failure, as an early institution of a brief course of complement blocking therapy in addition to HLH-targeted therapy may improve clinical outcomes in these patients.
- |||||||||| Gamifant (emapalumab) / SOBI
Review, Journal: Virus-triggered secondary hemophagocytic lymphohistiocytosis. (Pubmed Central) - Sep 16, 2021
The novel agent, emapalumab (an anti-IFN-γ monoclonal antibody) can be used to treat EBV-HLH cases to avoid the risk of secondary malignancy due to etoposide. Finally, HSCT is required for refractory EBV-HLH cases and can also be curative in some other cases.
- |||||||||| dexamethasone / Generic mfg.
Hemophagocytic Lymphohistiocytosis in a Liver Transplant Recipient (Shoreline Exhibit Hall) - Sep 8, 2021 - Abstract #ACG2021ACG_2282;
The patient was initially treated with dexamethasone followed by several cycles of etoposide, and finally emapalumab due to refractory disease...Treatment is with immunosuppression or chemotherapy. HLH is often fatal and prompt recognition and consultation with Hematology is necessary.Figure: Endoscopy photograph demonstrating denuded duodenal mucosa with bleeding.
- |||||||||| hydroxychloroquine / Generic mfg.
Acute Hepatitis in a Young Female with Lupus: It’s Not Always What You Think (Shoreline Exhibit Hall) - Sep 8, 2021 - Abstract #ACG2021ACG_2159;
Case Description/ A 21-year-old female with history of discoid lupus on hydroxychloroquine, cannabis hyperemesis syndrome, and morbid obesity was recently admitted to an outside hospital for shock of unclear etiology presumed to be due to culture-negative sepsis...She was discharged on prednisone 20mg oral daily for likely autoimmune hepatitis...She was started on treatment with pulse steroids, mycophenylate mofetil, anakinra, and plasma exchange without clear improvement...She was started on ruxolitinib without improvement and etoposide which was stopped due to neutropenia...Patient clinically improved on emapalumab-lzsg with improved inflammatory markers and normalization of liver enzymes...While AIH is often suspected in patients with rheumatologic disorders, other disorders such as HLH should be considered in a patient with elevated liver enzymes despite treatment. HLH is challenging to treat and alternative treatment options must be explored when patients are refractory to therapy.
- |||||||||| Gamifant (emapalumab) / SOBI, Jakafi oral (ruxolitinib) / Novartis, Incyte, Promacta (eltrombopag) / Novartis
Clinical, Review, Journal: The role of interferon-gamma and its signaling pathway in pediatric hematological disorders. (Pubmed Central) - Aug 11, 2021
Moreover, ruxolitinib, an inhibitor of JAK/STAT intracellular signaling, is currently being investigated for treating HLH...Eltrombopag, a small-molecule agonist of c-MPL, acts at a different binding site to IFN-γ and is thus able to circumvent its inhibitory effects. Ongoing trials will elucidate the role of IFN-γ neutralization in secondary HLH and future studies could explore this strategy in controlling hyperinflammation due to CAR T cells.
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI
Trial completion: A Study to Evaluate Emapalumab in Japanese Healthy Volunteers. (clinicaltrials.gov) - Aug 10, 2021
P1, N=8, Completed,
Ongoing trials will elucidate the role of IFN-γ neutralization in secondary HLH and future studies could explore this strategy in controlling hyperinflammation due to CAR T cells. Recruiting --> Completed
- |||||||||| Campath (alemtuzumab) / Sanofi
[VIRTUAL] Bridge to Transplantation for Refractory HLH (CHANNEL C) - Jul 31, 2021 - Abstract #ICBMT2021ICBMT_63;
Newer agents, including an interferon gamma blocking antibody (emapalumab) and JAK inhibitors (such as ruxolitinib) are emerging as HLH therapeutics...Second line therapies for HLH include alemtuzumab, which is effective, but produces prolonged immune ablation, which may com- plicate subsequent HSCT...Notably, HSCT outcomes after emapalumab treatment were unusually good, and require further study. Interferon gamma targeted therapies (either emapalumab or JAK inhibitors) are likely to be increasingly used for the treatment of HLH.
- |||||||||| Gamifant (emapalumab) / SOBI, Actemra IV (tocilizumab) / Roche, JW Pharma, Kineret (anakinra) / SOBI
Journal, Cytokine storm: Can emapalumab be life saving for refractory, recurrent, and progressive cytokine storm caused by COVID-19, which is resistant to anakinra, tocilizumab, and Janus kinase inhibitors. (Pubmed Central) - Jul 9, 2021
Emapalumab (Eb) is the human immunoglobulin G1 monoclonal antibody and is a potent and noncompetitive antagonist of IFN-γ. Eb can be life saving for cytokine storm caused by COVID-19, which is resistant to anakinra, tocilizumab, and JAK inhibitors.
- |||||||||| Gamifant (emapalumab) / SOBI
Review, Journal: Novel Therapeutic Approaches to Familial HLH (Emapalumab in FHL). (Pubmed Central) - Jun 24, 2021
Available data support the use of emapalumab for treatment of pHLH patients with refractory, recurrent or progressive disease, or intolerance to conventional therapy, recently, leading to FDA approval of the drug for these indications. Additional data are needed to define the role of emapalumab in front-line treatment or in combination with other drugs.
- |||||||||| Gamifant (emapalumab) / SOBI
Clinical, Journal: Emapalumab for adult and pediatric patients with hemophagocytic lymphohistiocytosis. (Pubmed Central) - Jun 22, 2021
The outcomes in patients who underwent transplantation compare favorably with those reported previously with either myeloablative or reduced-intensity conditioning regimens. The potential role of emapalumab in the treatment of secondary HLH and as a prevention of graft failure after HSCT deserves to be further assessed.
- |||||||||| Campath (alemtuzumab) / Sanofi
[VIRTUAL] CMV INFECTION WITH FATAL HEMOPHAGOCYTIC SYNDROME () - Jun 20, 2021 - Abstract #ICLLM2021ICLLM_129;
Allogeneic stem cell transplantation is another treatment option to refractory HLH patients. It may be hard to decide the treatment options in infection settings.
- |||||||||| Campath (alemtuzumab) / Sanofi
Clinical, Review, Journal: Clinical Management of Relapsed/Refractory Hemophagocytic Lymphohistiocytosis in Adult Patients: A Review of Current Strategies and Emerging Therapies. (Pubmed Central) - Apr 24, 2021
New drugs have been developed, such as emapalumab, ruxolitinib, and alemtuzumab, and they may be used as bridges to the curative HSCT...Mortality related to HSCT is mainly due to active HLH disease before HSCT and post HSCT complications. New drugs, such as empalumab, ruxolitinib, and alemtuzumab are interesting since these agents may be used as bridges to HSCT with increases in the numbers of patients proceeding to HSCT and survival rate.
- |||||||||| Gamifant (emapalumab-lzsg) / SOBI
Enrollment closed, Enrollment change, Trial completion date: A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH (clinicaltrials.gov) - Apr 20, 2021
P2/3, N=7, Active, not recruiting,
We report the use of emapalumab and cyclophosphamide in a patient with XLP-1 and EBV-associated HLH complicated by retinal vasculitis, resulting in clinical improvement. Recruiting --> Active, not recruiting | N=20 --> 7 | Trial completion date: Sep 2022 --> Nov 2021
- |||||||||| Campath (alemtuzumab) / Sanofi
[VIRTUAL] HSCT RESCUES MARROW FAILURE IN A PATIENT WITH LEUKEMIA AND PREVIOUSLY UNDIAGNOSED LIGASE IV SYNDROME () - Apr 14, 2021 - Abstract #ASPHO2021ASPHO_560;
Our case demonstrates successful rescue HSCT in a patient with cardiac failure, HLH, leukemia, and marrow aplasia, demonstrating that these patients may recover from significant co-morbid conditions that would otherwise preclude curative HSCT and tolerate a RIC regimen that facilitates engraftment. This research was performed at the Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Department of Pediatrics, Emory University, Atlanta, GA, USA
- |||||||||| Campath (alemtuzumab) / Sanofi
[VIRTUAL] HSCT RESCUES MARROW FAILURE IN A PATIENT WITH LEUKEMIA AND PREVIOUSLY UNDIAGNOSED LIGASE IV SYNDROME () - Apr 14, 2021 - Abstract #ASPHO2021ASPHO_559;
Case series have described successful HSCT with RIC preparative regimen for patients with ligase IV syndrome, though data are scant for the successful treatment of malignancy and recovery from organ dysfunction and HLH for this disease. Our case demonstrates successful rescue HSCT in a patient with cardiac failure, HLH, leukemia, and marrow aplasia, demonstrating that these patients may recover from significant co-morbid conditions that would otherwise preclude curative HSCT and tolerate a RIC regimen that facilitates engraftment.
- |||||||||| Gamifant (emapalumab) / SOBI
[VIRTUAL] PEDIATRIC REFRACTORY SECONDARY HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TREATED WITH EMAPALUMAB () - Apr 14, 2021 - Abstract #ASPHO2021ASPHO_461;
Objectives: Standard HLH therapy with dexamethasone and etoposide was established in 1994. This is the first report of successful use of emapalumab in refractory sHLH and serves as an encouraging rationale for future applications of the novel agent in a broader patient population with potential for improved outcomes in this devastating, rapidly progressive disease.
- |||||||||| Gamifant (emapalumab) / SOBI
[VIRTUAL] POST-HSCT OUTCOMES AFTER EMAPALUMAB TREATMENT IN PRIMARY HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS () - Apr 14, 2021 - Abstract #ASPHO2021ASPHO_312;
P=N/A, P2/
Emapalumab was administered on a background of dexamethasone 5-10mg/m2...Preparative regimens varied but included melphalan- (reduced intensity) and busulfan- (myeloablative) based regimens... IFNy neutralization with emapalumab permitted most patients with active pHLH to proceed to HSCT, and was associated with a favorable rate of engraftment, GvHD, and survival estimates.
- |||||||||| sildenafil / Generic mfg.
[VIRTUAL] Management of non-melanoma skin cancers (SCC, BCC, MCC) () - Mar 9, 2021 - Abstract #HOPA2021HOPA_72;
Disease state background and approaches to therapy will be reviewed, along with systemic treatment options including the role of immune checkpoint inhibitors and hedgehog inhibitors.emapalumab was the first medication approved for use by the FDA in those with refractory, recurrent, or progressive primary HLH or those intolerant to conventional therapy. This session will evaluate the management of those with primary HLH and adverse effects associated with treatment
- |||||||||| sildenafil / Generic mfg.
[VIRTUAL] Management of Primary Hemophagocytic Lymphohistiocytosis () - Mar 9, 2021 - Abstract #HOPA2021HOPA_68;
Recently, emapalumab was the first medication approved for use by the FDA in those with refractory, recurrent, or progressive primary HLH or those intolerant to conventional therapy. This session will evaluate the management of those with primary HLH and adverse effects associated with treatment.
- |||||||||| Gamifant (emapalumab) / SOBI
[VIRTUAL] POST-TRANSPLANT OUTCOMES OF CHILDREN WITH PRIMARY HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TREATED WITH EMAPALUMAB (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1494;
P=N/A, P2/
Emapalumab was administered on a background of dexamethasone 5-10 mg/m2...Preparative regimens varied but included melphalan- (reduced intensity) and busulfan- (myeloablative) based regimens... Neutralization of IFNγ with emapalumab permitted most patients with active pHLH to proceed to HSCT, and was associated with a favorable rate of engraftment, GvHD, and survival estimates. Clinical Trial Registry: NCT01818492; NCT02069899
- |||||||||| Gamifant (emapalumab) / SOBI
[VIRTUAL] POST-TRANSPLANT OUTCOMES OF CHILDREN WITH PRIMARY HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TREATED WITH EMAPALUMAB (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1493;
P=N/A, P2/
Emapalumab was administered on a background of dexamethasone 5-10 mg/m2...Preparative regimens varied but included melphalan- (reduced intensity) and busulfan- (myeloablative) based regimens... Neutralization of IFNγ with emapalumab permitted most patients with active pHLH to proceed to HSCT, and was associated with a favorable rate of engraftment, GvHD, and survival estimates. Clinical Trial Registry: NCT01818492; NCT02069899
- |||||||||| Gamifant (emapalumab) / SOBI
[VIRTUAL] POST-TRANSPLANT OUTCOMES OF CHILDREN WITH PRIMARY HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS TREATED WITH EMAPALUMAB (On-Demand Library) - Feb 4, 2021 - Abstract #EBMT2021EBMT_1492;
P=N/A, P2/
Emapalumab was administered on a background of dexamethasone 5-10 mg/m2...Preparative regimens varied but included melphalan- (reduced intensity) and busulfan- (myeloablative) based regimens... Neutralization of IFNγ with emapalumab permitted most patients with active pHLH to proceed to HSCT, and was associated with a favorable rate of engraftment, GvHD, and survival estimates. Clinical Trial Registry: NCT01818492; NCT02069899
- |||||||||| Gamifant (emapalumab) / SOBI
Journal: Estimating health state utilities in hemophagocytic lymphohistiocytosis. (Pubmed Central) - Jan 22, 2021
This study has estimated utility weights for seven different HLH related states which are based on detailed input from carers and physicians and have good face validity. There are few other options for collecting these data in an ultra-rare setting.
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