sirolimus / Generic mfg. - LARVOL DELTA

Outcomes of allogeneic hematopoietic stem cell transplantations (HSCT) for treatment of secondary primary malignancies arising post-autologous HSCT for multiple myeloma (ASH 2025) - "As conditioning regimens, 12 patients received Fludarabine/Busulfan, and 5 patients received Fludarabine/Melphalan...For GVHD prophylaxis, 6 patients received Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil (MMF); 9 patients received Thymoglobulin, Tacrolimus, and MMF; 1 patient received Thymoglobulin, Sirolimus, and MMF; 1 patient received Tacrolimus, Methotrexate, and Abatacept...Limitations of the outcome data include a relatively small population that underwent Allo-HCST, likely lower than the true number of patients with SPM following Auto-HSCT due to them either not returning to the center or opting against Allo-HSCT for treatment. This data adds to the collective understanding of the associated risks and benefits of Allo-HSCT for treatment of hematological SPM following Auto-HSCT for MM."

Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome • Oncology • Transplantation • TP53

Risk of relapse and mortality after non-myeloablative allogeneic stem cell transplant for Acute Myeloid Leukemia and myelodysplastic syndrome after fludarabine, cyclophosphamide, and 200 centigray total body irradiation with gvhd prophylaxis using post-transplant cyclophosphamide, sirolimus, and mycophenolate mofetil: Impact of maintenance chemotherapy (ASH 2025) - "Oral HMA agents were decitabine/cedazuridine (dec-c) 3d/28-day cycle, and oral azacitidine (Oral-Aza) for 14 days per 28-day cycle...A total of 32 (31%) patients received post-HSCT maintenance chemotherapy: infusional HMA (n=2), recombinant-Granulocyte Colony Stimulating Factor (rhGCSF)/decitabine (n=2), gilteritinib (n=7), dec-c (n=9), oral-aza (2), and venetoclax/Azacitidine (n=8)... NMA conditioning with Flu/Cy/2Gy TBI yields low NRM. Post-transplant maintenance therapy, particularly with HMAs and targeted agents, is associated with reduced relapse in intermediate-risk and adverse-risk TP53 wt AML and MDS. However, outcomes remain poor for patients with TP53 mu t AML and MDS, indicating an urgent need for alternative or intensified maintenance strategies in these high-risk groups."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation • FLT3 • TP53

Connecting pharmacokinetic exposure to immune reconstitution: A pilot study in HLA-haploidentical hematopoietic cell transplant recipients (ASH 2025) - "Post-transplant cyclophosphamide (PTCy) and mycophenolate mofetil (MMF) are commonly used in combination with tacrolimus or sirolimus as standard-of-care prophylaxis of graft-versus-host disease (GVHD) for allogeneic hematopoietic cell transplant (alloHCT). The findings also suggest that renal dysfunction may increase exposure to Cy and MMF metabolites. Our data supported the need for further research in larger cohorts to investigate the relationship between pharmacokinetic biomarkers and clinical outcomes."

Clinical • PK/PD data • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Nephrology • 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

Effect of GVHD prophylaxis on renal function and survival outcomes in allogeneic hematopoietic cell transplant patients with pre-existing renal impairment (ASH 2025) - "The advent of post-transplant cyclophosphamide (PTCy) permitted calcineurin inhibitor free graft-versus-host disease (GVHD) prophylaxis when combined with sirolimus (SIR), offering an HCT option that is less nephrotoxic...GVHD prevention regimens included PTCy/SIR, PTCy with tacrolimus (PTCy/TAC), TAC/SIR, and Tac with methotrexate (TAC/MTX)... Half of the patients with pre-existing RI, particularly those with eGFR <60, are at high risk of developing AKI post-HCT. While immunosuppression can significantly influence the risk of AKI, PTCy/SIR demonstrates a more favorable impact on renal function after HCT. These findings support the preferential use of PTCy/SIR as GVHD prophylaxis to reduce the risk of renal toxicity and associated morbidities, including need for dialysis post-HCT."

Clinical • Acute Kidney Injury • Cardiovascular • Diabetes • Diabetic Nephropathy • Graft versus Host Disease • Hematological Malignancies • Hypertension • Immunology • Metabolic Disorders • Nephrology • Renal Disease • Transplantation

Sirolimus plus low-exposure calcineurin inhibitors as an alternative gvhd prophylaxis in CNI-intolerant recipients: A retrospective study (ASH 2025) - "The cohort (10 males, 6 females) had a median age of 22.5 years (range 5–60). The regimen was initiated a median of 27.5 days post-transplant (range –2 to 114) and continued for a median of 53 days. Five patients (31.2%) received sirolimus and low-dose CNI alone, 10 (62.5%) received additional mycophenolate mofetil (MMF), and 1 patient received MMF plus anti-CD25 antibody."

Retrospective data • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease

Characterization and risk factors of transplantation-associated thrombotic microangiopathy (TA-TMA): Increased incidence associated to post-transplant cyclophosphamide (PTCy) prophylaxis. (ASH 2025) - "GVHD prophylaxis consisted of Tacrolimus-MTX in HLA-identical donor transplants with myeloablative conditioning regimen (27%), PTCy-based prophylaxis in haploidentical and other mismatched donor transplant with myeloablative conditionings (36%) and Tacrolimus-Sirolimus +/- mofetil mycophenolate in reduced-intensity conditioning regimen (36%). TA-TMA is associated with impaired OS, especially in those with organ involvement. PTCy is significant and independently linked to increased risk of TA-TMA, and co-occurrence of BK/JC cystitis or acute GVHD increased risk in these patients, suggesting that a closer monitorization should be implemented for them."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Cardiovascular • Graft versus Host Disease • Hematological Disorders • Hypertension • Immunology • Renal Disease • Thrombosis • Transplantation

Haploidentical hematopoietic stem cell transplantation with bussulfan, fludarabine and cyclophosphamide and total body irradiation 200cgy conditioning with post-transplant cyclophosphamide and peripheral blood stem cells as an alternative regimen to reduce graft rejection in sickle cell disease (ASH 2025) - "Graft-versus-host disease (GvHD) prophylaxis included PTCy (50 mg/kg on days +3 and +4), mycophenolate mofetil, and sirolimus. Our data support the feasibility and safety of haploidentical HSCT using busulfan, fludarabine, and cyclophosphamide RIC combined with PTCy and PBSC in adult SCD patients. This approach yielded sustained full donor chimerism with low incidence of severe GvHD and graft failure, offering a practical alternative in resource-limited settings where thiotepa is not easily available. Considering the ongoing challenges in donor availability and conditioning toxicity, further studies with larger cohorts and longer follow-up are warranted to confirm the durability of engraftment, late effects, and overall survival benefits."

Post-transplantation • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cytomegalovirus Infection • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Inflammation • Mucositis • Sickle Cell Disease • Transplant Rejection • Transplantation

Incidence, predictors and consequences of poor graft function after post-transplant cyclophosphamide (PTCy)-based allogeneic transplantation (ASH 2025) - "Patients who had PGF were more likely to be CMV positive, ABO incompatible, had microangiopathic hemolytic anemia (MAHA), acute GVHD, or had been treated with ruxolitinib or sirolimus. Although PGF significantly increased the risk of NRM, it had no impact on survival due to a corresponding decrease in relapse incidence. The positive effect of PGF on relapse reduction, which is independent of disease-related factors or the development of CMV reactivation or GVHD, deserves further study to understand the basis of this finding."

Post-transplantation • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation

The effect of belumosudil on the therapeutic monitoring of tacrolimus and sirolimus in allogeneic hematopoietic transplantation (ASH 2025) - "Azoles were used concomitantly in 51.5% of patients (35/68), with 65.7% (23/35) on posaconazole, 31.4% (11/35) on isavuconazole, and 2.9% (1/35) on voriconazole. Four patients started an azole after starting belumosudil (2 on posaconazole, 1 on fluconazole, and 1 on isavuconazole)... Our study confirms the drug interaction between TAC/SIRO, with the increase in TAC/SIRO levels more pronounced with the second level after starting belumosudil. However, severe toxicities were rarely seen. Based on our data, we recommend close TDM of TAC and SIRO, but no dose adjustment is warranted when belumosudil is added."

Acute Kidney Injury • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Movement Disorders • Nephrology • Transplantation • CYP3A4

The molecular landscape of patients with malignant histiocytosis (ASH 2025) - "Responders received a range of agents matched to their mutations: trametinib ( BRAFV600E, MAP2K1F53I, KRASQ16H/PTEND24G/EPHB1R327C, MAP2K1F53I/BCL2/CDKN1B/KIT, and CALR), dabrafenib + trametinib( BRAFG596R/KRASQ61H) , imatinib ( MAP2K1C121S/METG28881T/DUSP2G137D/HIST1H3BC97Y/GRIN2AS1216C and PTPN11/STK11/GNA11/JAK2) , chidamide + sintilimab( IDH2G515A/RHOAG50T/TET23344delC), sirolimus( PTEN/FGFR3/SETD2) and sorafenib ( BRAFD594G/KRASK117N/TP53). Despite this complexity, durable clinical responses to targeted agents are achievable, even in heavily mutated cases. These findings support the use of comprehensive genomic profiling in MH to identify therapeutic targets and guide precision treatment strategies."

Clinical • IO biomarker • Sarcoma • Solid Tumor • ADGRG6 • BCL2 • BCL6 • BRAF • CALR • CDK1 • CDKN1A • CDKN1B • CDKN2A • CDKN2B • CREBBP • CYTOR • DNMT3A • EGFR • FGFR3 • GNA11 • JAK2 • KRAS • MAP2K1 • NF1 • NRAS • NRF1 • PTEN • PTPN11 • SETD2 • STK11 • TET2 • TP53

Genome-wide CRISPR-Cas9 screening identified GPCR regulators RIC8A and PDCL as therapeutic targets in MLN with PDGFRB fusions (ASH 2025) - "Notably, pathway enrichment analysis suggested partial reliance on the mechanistic target of rapamycin (mTOR) pathway; however, pharmacologic inhibitors targeting mTOR failed to show selective efficacy...We identified RIC8A and PDCL as novel therapeutic targets in MLN-PDGFRB, potentially mediating crosstalk between RTK and GPCR signaling. Further in vivo validation and extension to other PDGFRB fusion partners are warranted to assess their translational relevance."

Eosinophilia • Hematological Malignancies • Lung Cancer • Solid Tumor • AGGF1 • EBF1 • EGFR • FGFR1 • PDGFRA • PDGFRB

KRAS mutations in histiocytic neoplasms: Mutation spectrum and response to MEK inhibition (ASH 2025) - "Regarding treatment, 57% (13/23) received MEKi, all initially cobimetinib, with 1 patient switched to trametinib due to progression. Of the 10 patients who did not receive MEKi, 1 was treated with vemurafenib for a concurrent BRAF p.V600E mutation; 3 remained on observation; 2 underwent surgery without recurrence; 3 received other systemic therapies including corticosteroid, methotrexate, rituximab, lenalidomide, or sirolimus; and 1 developed diffuse large B-cell lymphoma, treated with R-CHOP...A retrospective cohort described an ECD patient with p.K117N achieving PR on dabrafenib and trametinib with a concurrent BRAF mutation and another with p.G12A achieving PR on trametinib...As MEKi acts downstream, efficacy likely depends on the level of pathway activation conferred by specific KRAS variants. Larger studies are needed to confirm these observations and identify mutation-specific predictors to guide personalized treatment."

B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Langerhans Cell Histiocytosis • Lymphoma • Non-Hodgkin’s Lymphoma • Rare Diseases • ARAF • KRAS • MAP2K1 • NRAS • PIK3CA

Therapeutic Intervention of Corticospinal Tract Sprouting after Unilateral Traumatic Brain Injury via METTL1-Mediated tRNA m7G Modification. (PubMed, Mol Neurobiol) - "Critically, pharmacological inhibition of mTOR signaling with rapamycin diminishes the beneficial effects of METTL1 overexpression on axon outgrowth and CST sprouting. Our findings reveal a novel role for METTL1-mediated tRNA m7G modification in promoting neural repair and identify a potential therapeutic strategy for enhancing recovery after unilateral brain injury."

Journal • CNS Disorders • Vascular Neurology • METTL1

Warm autoimmune hemolytic anemia following epstein-barr virus reactivation: An uncommon clinical association (ASH 2025) - "His only medications were atenolol and atorvastatin, and he denied use of herbal supplements or over-the-counter drugs... Warm AIHA is most commonly idiopathic but can also occur secondary to various conditions, including rheumatologic diseases (e.g., systemic lupus erythematosus), chronic lymphoblastic leukemia, non-Hodgkin's lymphoma, certain medications (e.g., cephalosporins, piperacillin), and infections...Initial corticosteroid therapy varies widely during the first 72 hours, ranging from 1–2 mg/kg of prednisone every 8–12 hours to high-dose intravenous methylprednisolone (250–1000 mg/day)...In cases refractory to steroids, additional therapies such as mycophenolate mofetil or sirolimus may be considered. This case highlights a rare presentation of warm AIHA likely triggered by EBV reactivation, an association that is infrequently reported in the literature."

Clinical • Anemia • Autoimmune Hemolytic Anemia • Chronic Lymphocytic Leukemia • Cytomegalovirus Infection • Dyslipidemia • Epstein-Barr Virus Infections • Hematological Malignancies • Hepatology • Human Immunodeficiency Virus • Hypertension • Immunology • Infectious Disease • Inflammatory Arthritis • Leukemia • Lupus • Lymphoma • Non-Hodgkin’s Lymphoma • Pulmonary Disease • Rheumatology • Systemic Lupus Erythematosus • HP

Klippel-trenaunay syndrome with multifactorial anemia:successful management with sirolimus (ASH 2025) - "This case demonstrates: 1. KTS-associated vascular anomalies can precipitate life-threatening AIHA through antibody generation from chronic transfusion, necessitating rigorous blood group compatibility screening 2. mTOR inhibition Sirolimus– targeting PIK3CA-related pathogenesis – may concurrently ameliorate coagulopathy and vascular overgrowth."

Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immunology • Varicose Veins • PIK3CA

Complement inhibition in post-transplant IgA-mediated autoimmune cytopenia: A pediatric case report (ASH 2025) - "He developed cellular rejection one-year post-transplant and was treated with cyclosporine and MMF. At 7 years post-transplantation, he developed acute cellular rejection with third-degree heart block requiring pulse steroids and ATG, with subsequent transition to tacrolimus/sirolimus...Modification of immunosuppression is particularly challenging in the post solid organ transplant setting and can risk organ rejection requiring close collaboration with primary transplant team.⁵⁻⁶ In IgA-driven diseases like IgA nephropathy, complement inhibitors (e.g., eculizumab, narsoplimab) are gaining traction.⁷ This case adds to emerging evidence that complement modulation is a viable treatment pathway for refractory IgA-mediated AIHA, especially in the post-transplant setting. Eculizumab with prednisone was effective and well-tolerated in this case of IgA-mediated, rituximab-refractory AIHA post-heart transplant. Complement inhibition may represent a novel and safe therapeutic..."

Case report • Clinical • Post-transplantation • Autoimmune Hemolytic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • IgA Nephropathy • Immunology • Pediatrics • Renal Disease • Solid Organ Transplantation • Thrombocytopenia • Transplantation

Oxygen dependency as a critical regulatory axis affecting hematopoietic stem and progenitor cell function (ASH 2025) - "Rapamycin treatment to inhibit this mTORcprogram partially rescued low physiologic maintenance of potent HSC/HPC populations...Additionally, our O2-dependent transcriptomic map of HSCs/HPCs, reveals potential targets for altering cell function forclinical use. Our findings also have immediate translational implications in the context of expanded cordblood transplantation and other cell therapies that require ex vivo manipulation, like gene therapy,where O2 may be a critical factor in balancing ex vivo expansion while maintaining enough potent cellsfor early recovery and long-term hematopoietic durability."

Gene Therapies • CD34 • DDIT4 • ENO1 • LDHA

Serine/glycine starvation induces metabolic rewiring to uncover ATF4-driven therapeutic vulnerabilities in infant ALL (ASH 2025) - "An FDA-approved drug screencomparing S/G starved and non-starved cells revealed selective sensitivity to drugs targeting upstreampathways of ATF4 activation, such as Trazodone, Metformin and Rapamycin. This was supported by significant sensitivity of the cells to OxPhos inhibitors Metformin,Rotenone and Phenformin.In conclusion, S/G starved KMT2Ar cells rely on the ATF4-mediated ISR for survival. Therefore,repurposing drugs active upstream or downstream of ATF4 could open potential new, more effective, lesstoxic therapeutic windows in the treatment of infant leukaemia."

IO biomarker • Cardiomyopathy • Cardiovascular • Cognitive Disorders • Hematological Malignancies • Leukemia • Solid Tumor • AARS1 • ASNS • ATF4 • DDIT4 • KMT2A • MTHFD2 • SESN2 • SLC7A11 • TRIB3

Impact of fludarabine exposure on overall survival in patients receiving fludarabine/cyclophosphamide reduced intensity conditioning hematopoietic cell transplantation for hematologic malignancies (ASH 2025) - P1/2, P2 | "Graft-versus-host disease (GVHD) prophylaxis consisted of either alemtuzumab andcyclosporine (AC) or tacrolimus, methotrexate, and sirolimus (TMS). While our findings suggest that the current dosing approach is effective in preventingFlu overexposure, larger studies are needed to adequately assess the impact of Flu exposure on survivaloutcomes. Future prospective research is warranted to refine and validate optimal Flu exposure in theRIC alloHCT setting."

Clinical • Acute Myelogenous Leukemia • Chronic Lymphocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation

A US real-world analysis of demographics, transplant patterns, and survival outcomes in posttransplant lymphoproliferative disorder (PTLD) (ASH 2025) - "Other agents includedprednisone (6.2%), cyclosporine (5.8%), sirolimus (5.8%), dexamethasone (4.8%), azathioprine (3.1%),methylprednisolone (1.9%), everolimus (0.7%), ibrutinib (0.6%), ruxolitinib (0.6%), mycophenolate(0.3%), basiliximab (0.2%), and thymoglobulin (0.1%).Extranodal, bone marrow, and CNS involvement were present in 29.5%, 1.9%, and 1.8% of cases,respectively...Median OSfor heart transplant recipients was 2730 days, and for those who underwent HSCT, 2643 days.Regarding treatment, only 10 patients received bispecific antibodies (glofitamab, epcoritamab, ormosunetuzumab) and only 23 patients received CAR-T therapy. This study represents one of the largest real-world analyses of PTLD in adult patients inthe US, utilizing the TriNetX dataset spanning over three decades. This study represents one of the largest real-world analyses of PTLD in adult patients inthe US, utilizing the TriNetX dataset spanning over three decades. Improvement in OS in more recentyears..."

Clinical • Post-transplantation • Real-world • Real-world evidence • Bone Marrow Transplantation • Epstein-Barr Virus Infections • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Solid Organ Transplantation • Transplantation

IDH2 R172 mutation is associated with improved survival in IDH-mutated AML/MDS patients undergoing allogeneic stem cell transplantation: A geth-TC multicenter study (ASH 2025) - "In MVA, IDH2R172 independently predicted absence of relapse (HR 0.0; p<0.001).1-year NRM was 9.5% (95% CI, 3–15), ), and no significant predictors were identified in univariate analysis.The 100-day cumulative incidence (CI) of acute GvHD was 40% (95% CI, 30–49), significantly higher amongpatients receiving Tacrolimus–SirolimusGVHD prophylaxis compared with PTCy (HR 9.3; p<0.001).The 1-year CI of Chronic GvHD was 27% (95% CI, 18–37)...In this large multicenter series of IDH-mutated AML/MDS undergoing allo-HCT, the IDH2R172 mutationwas associated with a improved survival, a lower RI, and a higher GRFS. Whether this reflects favorabledisease biology or increased sensitivity to graft-versus-leukemia effects following allo-HCT remainsunclear. Our findings may have direct clinical implications for risk stratification and transplant decision-making and warrant prospective validation."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation • BCOR • IDH1 • IDH2 • NPM1

ZFP36L2 as a promising therapeutic target in acute graft-versus-host disease: Evidence from murine models and clinical validation (ASH 2025) - "Restoring ZFP36L2 experssion by rapamycin could recalibrate Treg-mediated immunosuppression in aGVHD. However, the precise molecular mechanisms underlyingZFP36L2-mediated immunoregulation require further investigation."

Preclinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • CD8 • FOXP3 • GAPDH • PCLAF • ZFP36 • ZFP36L1

Non-genotoxic conditioning to increase donor chimerism levels in a mismatched murine transplant model for sickle cell disease (ASH 2025) - "The recently FDA-approved gene therapies Lyfgenia and Casgevy also employ MAC...We first investigated theefficiency of CD45- and CD117-sap as conditioning agents compared to classical total body irradiation(TBI) and busulfan. Our base conditioning regimen also included post-transplant cyclophosphamide (PT-Cy) and sirolimus...HPLC analysis at 20 weeks post-HCT showed that recipients conditioned with CD117-sapexhibited donor-derived Hb, while recipients conditioned with 200cGy TBI showed mixed donor/recipientHb. Furthermore, BM analysis at 24 weeks post-HCT also showed a significant increase in the frequenciesof donor-derived short- (52.50±5.67% vs 4.80±2.40%, p=0.0010) and long-term (43.28±5.79% vs0.79±0.79%, p=0.0016) hematopoietic stem cells in CD117-sap conditioned recipients, which furtherconfirms the successful long-term engraftment of CD117-sap conditioned recipients.In summary, CD117-sap, in combination with anti-thy1.2, PT-Cy, and sirolimus, is..."

Preclinical • Anemia • Gene Therapies • Genetic Disorders • Hematological Malignancies • Sickle Cell Disease • Transplantation • KIT • PTPRC • THY1

Real world evidence of treatment patterns in hereditary hemorrhagic telangiectasia (ASH 2025) - "Treatment examinedincluded bevacizumab, pazopanib, pomalidomide, aminocaproate, tranexamic acid (TXA), octreotide,lanreotide, thalidomide, tacrolimus, sirolimus, tamoxifen and raloxifene...The majority of HHT patients were treated withferrous sulfate (n=910), followed by iron sucrose, ferrous gluconate, ferric carboxymaltose, iron dextran,and ferric derisomaltose...While 8% of patients treated with TXA had VTE, the data does not support a definitiveconclusion of increased VTE in patients treated with anti-fibrinolytics. To further elucidate treatmentpractices in HHT, a multi-center approach incorporating review of medical records would providevaluable insights into real-world practices."

Clinical • HEOR • Real-world • Real-world evidence • Anemia • Aplastic Anemia • Cardiovascular • Cerebral Hemorrhage • CNS Disorders • Gastrointestinal Disorder • Hematological Disorders • Hypertension • Renal Disease • Venous Thromboembolism