cyclosporine / Generic mfg. - LARVOL DELTA

U.S. mortality trends in acquired aplastic anemia, 1999–2021: A joinpoint analysis using CDC wonder data (ASH 2025) - "Survival has improved dramatically since immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (HSCT) became standard, and after eltrombopag was added to therapy...This coincides with widespread adoption of horse antithymocyte globulin plus cyclosporine regimens, leading to reported 70–80 % hematologic response rates and improved survival...The study underscores the translational impact of clinical innovations on population-level outcomes. Continued surveillance is crucial as newer agents, biomarkers, and transplantation strategies emerge."

Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome

The clinical observation of treating autoimmune cytopenias after allogeneic hematopoietic stem cell transplantation in children by immunoinduction therapy (ASH 2025) - "In all cases, corticosteroids were treated with or without IVIG and rituximab for 1 to 5 weeks, and then Immunosuppressants such as cyclosporine or tacrolimus etc, are reduced and discontinued, combined with or without PD1 inhibitors and DLI were used to treat AICs. At a median follow-up of 20(2-62) months after discontinuation of treatment, none of the children had recurrence. The immunoinduction therapy of AICs after allogeneic hematopoietic stem cell transplantation is safe and effective."

Clinical • Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Hurler Syndrome • Immunology • Neutropenia • Primary Immunodeficiency • Transplantation

Long-term HIV remission of a perinatally infected individual, following a hematopoietic cell transplantation from a CCR5Δ32 homozygous donor for multiple myeloma: The kyiv patient (ASH 2025) - "Complete remission (CR) was achieved after one cycle of melphalan/prednisone and local radiotherapy, but an abdominal relapse occurred six months later. Despite subsequent lines of therapy (bortezomib(bort)/lenalidomide(lena)/dexamethasone(dexa) and bendamustine/bort/dexa), the extramedullary multiple myeloma (MM) progressed. Remission was achieved after dexa/thalidomide/cisplatin/doxorubicin/cyclophosphamide/etoposide (DT-PACE) and an autologous HCT performed on 1/2021...Allogeneic HCT was performed in 8/2022 after fludarabine/melphalan conditioning and anti-thymocyte globulin from a CCR5-Δ32homozygous, unrelated HLA-matched donor; cyclosporine/methotrexate was utilized as graft-versus-host diseas e (GVHD) prophylaxis... To our knowledge, this represents the first report of ART-free HIV RNA suppression following allogeneic HCT with a CCR5Δ32homozygous donor in an individual perinatally infected with HIV. Despite the differences in the latent reservoir and the mechanism..."

Clinical • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Human Immunodeficiency Virus • Immunology • Infectious Disease • Multiple Myeloma • Plasmacytoma • Transplantation • CCR5 • CD4 • CD8

Demographic and clinical risk factors for renal complications and associated non-relapse mortality in hematopoietic stem cell transplantation (ASH 2025) - "Antithymocyte globulin was used in 65.4% of conditioning regimens, and cyclosporin was the primary graft-versus-host disease (GvHD) prophylaxis (94.6%)...Renal complications represent major contributors to NRM in HSCT patients. These findings highlight the need for early intervention and integrated renal care in HSCT management."

Clinical • Acute Graft versus Host Disease • Acute Kidney Injury • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Kidney Disease • Graft versus Host Disease • Immunology • Infectious Disease • Nephrology • Transplantation

Long-term update on safety and efficacy of low-dose post-transplant cyclophosphamide in the HLA-mismatched unrelated transplant setting (ASH 2025) - "Materials and Methods A prospective, single-centre study was conducted to assess the safety and efficacy of reduced-dose PTCy (15 or 25mg/kg), combined with low-dose alemtuzumab (5mg/day for 2 days) and cyclosporin, as GvHD prophylaxis in 9/10 mismatched unrelated (MMUD) peripheral blood stem cell transplant (PBSCT) setting (study group). Moreover, the ability to tailor PTCy dosing based on disease and relapse risk could allow for a personalized approach to GvHD prevention. These results warrant validation in larger prospective, randomized studies and exploration of novel PTCy-based combinations, potentially paving the way for new standards in GvHD prophylaxis."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Transplantation

Early detection of emerging safety signals in GVHD prophylaxis agents using a 12-year faers "slope-watch" approach (ASH 2025) - "In addition to tacrolimus and cyclosporine, contemporary prophylaxis regimens employ sirolimus and everolimus, mycophenolate mofetil, methotrexate, post-transplant cyclophosphamide (PTCy), JAK inhibitors (ruxolitinib, baricitinib, tofacitinib), co-stimulation blockade with abatacept, gut-homing integrin antagonism via vedolizumab, and the ROCK2 inhibitor belumosudil. Sequential IC-Δ offers a robust, forward-looking pharmacovigilance framework that identifies adverse event acceleration 12–24 months before regulatory action. By emphasizing directional change and incorporating a simple confidence approximation, this approach enhances early signal detection in large safety datasets. Integration with electronic health records, regulatory pipelines, and interactive dashboards could further streamline horizon scanning and improve patient safety in GVHD prophylaxis."

Clinical • Bone Marrow Transplantation • CNS Disorders • Cytomegalovirus Infection • Dyslipidemia • Graft versus Host Disease • Hemophagocytic lymphohistiocytosis • Hepatology • Immunology • Infectious Disease • Metabolic Disorders • Pneumonia • Rare Diseases • Respiratory Diseases • Septic Shock

Line-of-therapy stratified efficacy of belumosudil in cgvhd : Real-world evidence (ASH 2025) - "For concomitant treatments, cyclosporine was more frequent in patients with 1 prior LOT (73.3% vs. 22.2%, P=0.033), whereas tacrolimus was more common in patients with ≥2 prior LOTs (13.3% vs. 66.7%, P=0.021). No statistically significant differences were observed for other concomitant treatments, including corticosteroid (73.3% vs. 33.3%, P=0.092), mycophenolate mofetil (80.0% vs. 66.7%, P=0.635) and JAK inhibitor (33.3% vs. 44.4%, P=0.678)... In this real-world analysis dedicated to pretreated cGVHD, belumosudil achieved 95.8% (23/24) ORR in overall cohort, with 100% ORR specifically in second-line therapy (15/15). The 88.9% ORR in patients with ≥2 prior LOTs (8/9), 41.7% steroid reduction, and no grade≥3 AEs support its prioritization for early second-line use."

Clinical • HEOR • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

Effectiveness of belumosudil in acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT): A real-world observational study (ASH 2025) - "All patients received myeloablative conditioning, with 75% of patients administered the fludarabine plus modified TBI regimen incorporating craniospinal, marrow, and nodal targets. Standard GVHD prophylaxis was cyclosporine/MMF/methotrexate + anti-CD25/ATG/ALG...Conclusion : This study provides the first disclosed real-world evidence of belumosudil in acute GVHD, demonstrating unprecedented 100% ORR (75% cases with a complete response) with rapid median time to response of 13.5 days and well tolerated. Despite the inherent limitations of this pilot investigation, these findings warrant validation in pivotal multicenter trials."

Clinical • Observational data • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Dermatology • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Respiratory Diseases • Transplantation • CD80 • CD86 • IL17A • IL21 • STAT5

Exploring the efficacy and safety of low-dose ATG combined with fractionated low-dose ptcy in preventing gvhd after allogeneic hematopoietic stem cell transplantation (ASH 2025) - "Background In recent years, growing evidence has demonstrated the potential of anti-thymocyte globulin (ATG) combined with post-transplant cyclophosphamide (PTCY) as an effective strategy for graft-versus-host disease (GVHD) prophylaxis...The GVHD prophylaxis regimen consisted of low-dose ATG (2.5 mg/kg on days -2 and -1), fractionated low-dose PTCY (25 mg/kg on days +3 and +4), cyclosporine (2.5 mg/kg /day starting from day +5), and mycophenolate mofetil (0.5 g twice daily from day +5)...Conclusion GVHD prophylaxis with low-dose ATG combined with fractionated low-dose PTCY in allogeneic hematopoietic stem cell transplantation demonstrated excellent tolerability, with a remarkably low incidence of grade III-IV GVHD and absence of cardiac adverse events. However, due to the small sample size and short follow-up, further prospective, large-scale, multicenter randomized controlled trials are needed to validate the superiority of this regimen."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Mucositis • Stomatitis • Transplant Rejection • Transplantation

Single vs double cord blood transplantation: Improved engraftment with comparable efficacy (ASH 2025) - " Between April of 2006 and February of 2025, 315 patients with malignant disease underwent their first single (sCBT) or double CBT (dCBT) using a myeloablative conditioning regimen with FLU 75 mg/m2, TBI 13.2 Gy, CY 120 mg/kg (n=182) or FLU 150 mg/m2, TBI 4 Gy, CY 50 mg/kg, Thiotepa 10 mg/kg (n=47) or Treo 42 g/m2, FLU 150 mg/m2, TBI 2 Gy (n=86)...Graft-versus-host-disease (GVHD) prophylaxis included Cyclosporine and Mycophenolate Mofetil for all patients... In our study, sCBT was not inferior to dCBT with outstanding clinical outcomes. In fact, sCBT was associated with significantly faster neutrophil and platelet recovery. These findings encourage the use of sCBT in patients with an adequate cell dose, offering the potential to substantially reduce the cost of CBT without compromising efficacy."

Clinical • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Transplantation • CD34 • HLA-B • HLA-DRB1

A novel reduced-toxicity conditioning regimen with busulfan/fludarabine/cyclophosphamide/anti-thymocyte globulin for severe aplastic anemia (ASH 2025) - "The conditioning regimen comprised Bu (0.8 mg/kg every 6 hours, 1 day, weight-adjusted dose for pediatric patients), Flu (30 mg/m²/day, 4 days), Cy (500 mg/m²/day, 4 days), and ATG with Thymoglobulin 2 mg/kg/day, 4 days in 3 patients or ATG-Fresenius 5 mg/kg/day, 4 days in 7 patients. Graft-versus-host disease (GVHD) prophylaxis included cyclosporine and mycophenolate mofetil for all patients, supplemented with either: short-term methotrexate(MTX, +1d 15mg/m², +4d, +8d, +11d 10mg/m², n=2); only CD25 monoclonal antibody(Basiliximab, +3d 20mg, n=1); or both(MTX as above plus Basiliximab, +3d 20mg, n=2; or MTX as above plus Recombinant humanized anti-CD25 monoclonal antibody, +4d, +8d, 1mg/kg, n=5)...Majority of the patients are with good quality of life. Longer follow-up and larger series are needed to evaluate fertility and transplant outcomes with current protocol."

Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

Eravacycline in allogeneic stem cell transplant recipients: A real-world study of drug-drug interactions with cyclosporine/tacrolimus and anti-infective efficacy (ASH 2025) - "Eravacycline, a novel synthetic tetracycline, shares the Cytochrome P450 3A4 (CYP3A4) metabolic pathway with CNIs, yet comprehensive data on their potential pharmacokinetic interactions are scarce...Subgroup analysis revealed that when co-administered azoles and letermovir, no significant changes in C/D ratio were observed between pre-treatment and during treatment both in cyclosporine group (n=18) and tacrolimus group (n=7)... This study presented the first clinical evidence of drug-drug interaction between eravacycline with CNIs in allo-HSCT, characterized by increased CNI exposure during eravacycline administration and delayed post-cessation exposure elevation. Intensified CNI monitoring and stepwise dose adjustments are recommended throughout and after eravacycline therapy."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cardiovascular • Graft versus Host Disease • Hematological Disorders • Hypertension • Immunology • Infectious Disease • Transplantation • CYP3A4

Myelodysplastic syndrome in Tanzania; The current status of management (ASH 2025) - "Erythropoiesis-stimulating agents, lenalidomide, and cyclosporine were administered to 4.9%, 6.6%, and 8.2% of patients, respectively. Azacitidine, a hypomethylating agent, was offered to only 1.6% of patients...Supportive care remains the mainstay of management, with limited use of disease-specific therapies. This emphasizes the pressing need to strengthen diagnostic infrastructure and make treatment options available to align with global standards and improve the outcomes of MDS patients in low-resource settings."

Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome

Consolidation of dual MAPK inhibitor therapy by allograft in histiocytic sarcoma: A report of two cases (ASH 2025) - "Transplant proceeded using fludarabine 150mg/m 2 and melphalan 140mg/m 2 with post-grafting cyclophosphamide, mycophenolate mofetil and tacrolimus for graft-versus-host disease (GvHD) prophylaxis...He remains well on dabrafenib and trametinib in CMR at 16-months post-transplant...To consolidate this response, allograft from a haploidentical donor was performed using fludarabine 150mg/m 2 and melphalan 140mg/m 2 with post-grafting cyclophosphamide and ciclosporin for GvHD prophylaxis... These 2 cases suggest that allograft is feasible on dual MAPK inhibitor therapy and may have potential efficacy as consolidation for high-risk visceral HS. Withdrawal of inhibitors and longer follow up are required to determine the durability of remission with this approach."

Clinical • Cough • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Immunology • Infectious Disease • Respiratory Diseases • Sarcoma • Solid Tumor • Tuberculosis • BCL6 • CCND1 • CD14 • CD163 • CD1a • KRAS • MAP2K1

A tale of two lymphomas: A rare case of transformative post-transplant lymphoproliferative disorder (ASH 2025) - "Our 61-year-old patient underwent a living-unrelated renal transplant in 2014 for focal segmental glomerulosclerosis and was maintained on mycophenolate mofetil (MMF) and cyclosporine (CsA)...MMF was discontinued, and he received four weekly doses of rituximab (375 mg/m²), achieving complete remission (CR) and was consolidated with four additional Rituximab doses by Jan 2024...After improvement of liver and kidney function, therapy was escalated to brentuximab vedotin (BV) with cyclophosphamide, doxorubicin, and prednisone (CHP), given his CD30 expression...After detailed goals-of-care discussions, he and the care team elected to proceed with additional therapy with EPOCH (etoposide, prednisone, vincristine, cyclophosphamide, and doxorubicin), since he already received azacitidine, romidepsin, and BV-CHP...His care underscores the importance of multidisciplinary collaboration, patient-centered decision-making, and longitudinal follow-up. He has been referred to our..."

Clinical • IO biomarker • Post-transplantation • Bone Marrow Transplantation • Chronic Kidney Disease • Epstein-Barr Virus Infections • Febrile Neutropenia • Focal Segmental Glomerulosclerosis • Follicular Lymphoma • Glomerulonephritis • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Hepatology • Immunology • Infectious Disease • Liver Failure • Lymphoma • Nephrology • Peripheral T-cell Lymphoma • Rare Diseases • T Cell Non-Hodgkin Lymphoma • Transplantation • BCL6 • CD21 • CD4 • CD5 • CD7 • ICOS • JAK1 • PD-1 • RHOA • TET2 • TNFRSF8

Allogeneic hematopoietic stem cell transplantation in treatment of Shwachman- diamond syndrome: A Report of 25 Pediatric cases from a Single center (ASH 2025) - "All patients used myeloablating conditioning.The regimen of the case with AML consisted of busulfan, fludarabine, cytarabine, cyclophosphamide and anti-thymocyte globulin(ATG)...All were given cyclosporine and mycophenolate mofetil to prevent graft-versus- host disease(GVHD). Balliximab was given at +1 day to the patients whose donor was MMRD and methotrexate was given at +1,+4,+7,+11 days to the patients whose donor was MMUD or UCB...Melphalan was administered before donor peripheral blood stem cell infusion, resulting in negative MRD... Allo-HSCT is an effective treatment for SDS complicated with severe hematological abnormalities.Compared with MMRD and MMUD HSCT, the incidence of GVHD in CBT is lower. MDS complicated with both complex karyotype and Tp53 mutation was associated with an extremely poor prognosis."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Neutropenia • Palliative care • Pediatrics • Thrombocytopenia • Transplantation • TP53

Efficacy of combination immunotherapy of eltrombopag, ATG and cyclosporine in aplastic anaemia (ASH 2025) - "Triple immunotherapy with Eltrombopag, ATG, and Cyclosporine shows a promising and well-tolerated first-line treatment for severe AA as it has faster, deeper, and more sustained hematologic responses with minimal side effects. These findings support the incorporation of Eltrombopag into frontline management for patients with AA. Large scale clinical trials are required to assess durability of response and late clonal risks."

Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders

Trial in progress: Romiplostim N01 combined with immunosuppressive therapy in patients diagnosed with non-severe aplastic anemia, a Phase II study (ASH 2025) - P2 | "However, clinical data on romiplostim in NSAA remain limited, and no studies to date have evaluated the efficacy and safety of Romiplostim N01 in this population.Study Design and Methods We initiated a prospective, open-label, multi-centre, single-arm phase II trial (ChiCTR2500096280), conducted across multiple regions in China, to evaluate the efficacy and safety of Romiplostim N01 combined with cyclosporine or tacrolimus in patients diagnosed with NSAA and severe thrombocytopenia (platelet counts <30×10⁹/L)...Major eligibility criteria are: age ≥ 16, confirmed NSAA diagnosis, ECOG performance status of 0–2, QT interval <460 ms on electrocardiogram, adequate hepatic and renal function, prior TPO-RA recipients (including eltrombopag, hetrombopag, or avatrombopag) must complete a 1-month washout period before enrollment.Endpoints The primary endpoint is the overall hematologic response rate at weeks 12 and 24...A total of 40 subjects are..."

Clinical • P2 data • Anemia • Aplastic Anemia • Thrombocytopenia

Optimizing PNH treatment with the complement inhibitor pegcetacoplan: A case report (ASH 2025) - "The current treatment landscape includes 6 approved complement cascade inhibitors: 3 C5 inhibitors (eculizumab, ravulizumab, crovalimab), 1 C3/C3b inhibitor (pegcetacoplan), 1 factor B inhibitor (iptacopan), and 1 factor D inhibitor used as add-on treatment (danicopan)...Concomitant medications included apixaban, penicillin, and folic acid. In November 2020, her platelets count declined, and a bone marrow evaluation was diagnostic for moderate aplastic anemia (55-65% cellularity for age) and she was started on eltrombopag and cyclosporin. Despite ravulizumab and eltrombopag treatments, the patient developed significant anemia related to extravascular hemolysis (hemoglobin, 5.7 g/dL; LDH, 495 U/L; C5, 26.1 mg/dL [high]; complement hemolytic activity 50 [CH50], 7 U/mL [low])...After receiving both pegcetacoplan and iptacopan for 1 week and rivaroxaban 10 mg once daily for 48 hours, pegcetacoplan treatment ended on May 16, 2024... For this patient with PNH, the..."

Case report • Clinical • Anorexia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Meningococcal Infections • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Successful retreatment of PNH with switch to ravulizumab and add-on danicopan after experiencing breakthrough hemolysis on pegcetacoplan: A case report (ASH 2025) - "Terminal complement inhibition with C5 inhibitors ravulizumab (Rav), the standard of care for PNH treatment, and eculizumab (Ecu) have provided effective control of terminal complement activity and IVH and decreased rates of thrombosis...The patient was treated intermittently with antithymocyte globulin and/or cyclosporine for ~5 years while receiving red blood cell transfusions... Hb, 11.8 g/dL; LDH, 351 U/L). The patient restarted Rav with 2 loading doses 2 weeks apart. The patient experienced 1 mild episode of BTH ~4 months after restarting Rav (Hb, 9.9 g/dL; LDH, 382 U/L), resolving rapidly with her scheduled dose of Rav."

Case report • Clinical • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

Qualitative assessment of predictors of response to immunosuppressive therapy in aplastic anemia: A systematic literature review (ASH 2025) - "Recommended treatments include hematopoietic stem cell transplantation or immunosuppressive therapy (IST) with horse antithymocyte globulin and cyclosporine (double therapy) ± eltrombopag (triple therapy). This SLR combines all findings to date and adds genetic and molecular predictors to general AA knowledge. Further, more in-depth analyses are planned to elucidate details regarding the identified predictors, drill down on conflicting findings such as those related to PNH clones and explore additional predictors."

Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • B3GAT1 • CD8 • IFNG • TGFB1

A case for CD38 targeted therapy in multi-refractory immune thrombocytopenia (ASH 2025) - "Initial treatment with high-dose dexamethasone and IVIG was ineffective. He subsequently received rituximab, eltrombopag and avatrombopag...During weeks 8–14, he was treated with romiplostim, a second steroid pulse, mycophenolate, cyclophosphamide, and cyclosporine without response...While this study is ongoing, we present this case to highlight that daratumumab can be associated with a rapid platelet recovery in multi-refractory ITP, was safe and well tolerated with fostamatinib and danazol, and produced a durable response after a finite treatment course (12 weekly doses). In conclusion, for patients with prolonged, severe ITP unresponsive to conventional treatments, daratumumab offers a rational, mechanism-based intervention. Our case contributes to growing evidence supporting anti-CD38 immunotherapy in ITP and underscores the need for clinical trials to more broadly evaluate other plasma cell directed therapies for the treatment of ITP."

Clinical • Autoimmune Hemolytic Anemia • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Immune Thrombocytopenic Purpura • Immunology • Lymphoma • Non-Hodgkin’s Lymphoma • Thrombocytopenia • Thrombocytopenic Purpura

A single center review of secondary hemophagocytic lymphohistiocytosis treatment and outcomes. (ASH 2025) - "Initial therapy was malignancy-specific chemotherapy (39%), HLH-94 protocol based therapy (30%), steroids alone (23%), and anakinra (2%) while others were not treated (6%)...Overall, 46% of patients required several lines of treatment and were subsequently treated with several additional agents in the second line setting including malignancy directed therapy, ruxolitinib, alemtuzumab, eculizumab, IVIG, cyclosporine, tacrolimus and rituximab...To improve timely diagnosis and treatment decisions, we, like other centers, are implementing electronic medical record order sets for HLH diagnosis and management and organizing an HLH expert panel to provide timely case reviews and treatment recommendations. In memory of our esteemed colleague and co-author Stephen Njau, MD"

Clinical • Review • Dyslipidemia • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Hypertriglyceridemia • Immunology • Infectious Disease • Rare Diseases • Solid Tumor • IL2RA • ISG20

Efficacy and safety of iptacopan in paroxysmal nocturnal hemoglobinuria (PNH): A real-world study of 35 patients from anhui, China (ASH 2025) - "All received iptacopan (200 mg twice daily), with 12 patients also taking cyclosporine and 8 receiving testosterone undecanoate...Safety profile: Mild rash occurred in 6 patients (17.1%), all on concurrent prednisone; symptoms resolved after stopping prednisone, with no recurrence on iptacopan monotherapy...Responses are particularly robust in classical PNH and younger patients, while those with BMF may benefit from early combination therapy. Its favorable safety profile supports long-term use, offering valuable insights for individualized PNH management, particularly in Asian populations."

Clinical • Real-world • Real-world evidence • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CD59 • CFB

Efficacy of cyclosporine and antithymocyte or antilymphocyte globulin in the management of aplastic anemia: A systematic review and meta-analysis (ASH 2025) - "A total of ten studies (n=1,182) were identified, from which selected studies qualified for meta-analysis based on comparator arm compatibility. CsA plus ATG/ALG demonstrated a pooled remission rate of 66% (95% CI: 0.59-0.73; I²=28%) and a five-year overall survival rate of 80% (95% CI: 0.73-0.86; I²=0%). Treatment was associated with a relapse rate and infection rate of 62% (95% CI: 0.54-0.68; I²=0%) and 46% (95% CI: 0.24-0.69; I²=89.3%), respectively."

Retrospective data • Review • Anemia • Aplastic Anemia • Hematological Disorders • Infectious Disease