This dataset contains summary data visualizations and clinical data from a broad sampling of over 200 acute myeloid leukemias from 200 patients. The data was gathered as part of the PanCancer Atlas initiative, which aims to answer big, overarching questions about cancer by examining the full set of tumors characterized in the robust TCGA dataset. The clinical data includes mutation count, information about mutated genes, patient demographics, disease status, tumor typing, and chromosomal gain or loss. The data set also includes copy-number segment data downloadable as .seg files and viewable via the Integrative Genomics Viewer.

Patient demographics, clinical data and cytogenetic changes in AML patients with/without IDH1 mutations.

TCGA Acute Myeloid Leukemia. Source data from GDAC Firehose. Previously known as TCGA Provisional. This dataset contains summary data visualizations and clinical data from a broad sampling of 200 carcinomas from 200 patients. The data was gathered as part of the Broad Institute of MIT and Harvard Firehose initiative, a cancer analysis pipeline. The clinical data includes mutation count, information about mutated genes, patient demographics, sample type, disease code, Abnormal Lymphocyte Percent, Atra Exposure, Basophils Cell Count, Blast Count, Cytogenetic abnormality type, and FAB. The dataset includes Next-Generation Clustered Heat Maps (NG-CHM) viewable via an embedded NG-CHM Heat Map Viewer, provided my MD Anderson Cancer Center, which provides a graphical environment for exploration of clustered or non-clustered heat map data. The data set also includes copy-number segment data downloadable as .seg files and viewable via the Integrative Genomics Viewer.

BackgroundDisease-specific factors associated with decreased quality of life (QoL) in patients with leukemia have not been studied in a large-scale, global, observational study.MethodsThis cross-sectional study used the validated Hematological Malignancy Patient Reported Outcomes (HM-PRO) questionnaire to assess the impact of leukemia subtype, age, sex, and years living with the disease on QoL of patients with leukemia.ResultsOverall, 2,628 patients responded: 45.7% had chronic lymphocytic leukemia (CLL), 34.0% had chronic myeloid leukemia (CML), 11.8% had acute myeloid leukemia (AML), and 3.5% had acute lymphoblastic leukemia (ALL). HM-PRO scores differed significantly between leukemia subtypes (p

The Supplementary Tables file includes Tables S1 to S15. Table S1: Frequencies of CD34+CD38- LSCs within primary AML patient samples. Table S2: Immunopeptidomics cohort overview. Table S3: Patient characteristics of AML immunopeptidomics cohort. Table S4: AML- and AML/LSC-associated HLA class I targets. Table S5: AML/LSC shared HLA class I-presented antigens. Table S6: AML- and AML/LSC-associated HLA class II peptide, protein, and hotspot targets. Table S7: AML/LSC shared HLA class II-presented peptide targets. Table S8: Recurrent AML mutations included in neoepitope screening. Table S9: Characteristics of AML patient samples used for T cell-based assays. Table S10: Characteristics of HV samples used for T cell-based assays. Table S11: Immunopeptidome diversity according to demographics and disease characteristics. Table S12: Impact of demographics and disease characteristics on patient outcome in the immunopeptidome survival analysis group. Table S13: Patient characteristics of immunopeptidome survival analysis group. Table S14: Patient characteristics of immune response survival analysis group. Table S15: Impact of demographics and hematopoietic stem cell transplantation on patient outcome in the immune response survival group.

Abbreviations: AVP, arginine vasopressin; CSF, cerebrospinal fluid; ALL, acute lymphoblastic leukemia; AML, acute myeloblastic leukemia1indicates CSF collected from lumbar puncture2indicates CSF collected from left ventricle3indicates CSF collected from the cisterna magna.Patient demographics and medical characteristics.

Acute Myeloid Leukemia Drugs Market Outlook

According to our latest research, the global acute myeloid leukemia (AML) drugs market size reached USD 2.95 billion in 2024, reflecting the increasing prevalence of AML and the introduction of novel therapies. The market is expected to grow at a robust CAGR of 11.7% from 2025 to 2033, reaching an estimated USD 8.19 billion by 2033. This remarkable growth is primarily driven by advancements in targeted therapies, rising R&D investments, and a surge in awareness and early diagnosis of AML worldwide.

Several critical growth factors are fueling the expansion of the acute myeloid leukemia drugs market. Firstly, the rising incidence of AML, particularly among the aging population, is a significant driver. AML is most common in adults over 60, and as the global demographic shifts toward an older age profile, the patient pool is expanding. Additionally, improvements in diagnostic capabilities have led to earlier detection and more precise classification of AML subtypes, allowing for tailored treatment regimens. This has increased the demand for both conventional and novel drug therapies, as clinicians strive to improve survival rates and quality of life for patients.

Another important factor contributing to market growth is the rapid pace of innovation in drug development. Pharmaceutical companies and research institutions are investing heavily in the development of targeted therapies and immunotherapies, which offer improved efficacy and reduced side effects compared to traditional chemotherapy. The approval and commercialization of drugs such as FLT3 inhibitors, IDH inhibitors, and antibody-drug conjugates have transformed the treatment landscape. These advancements have not only increased the range of options available for patients but have also driven up the value of the market as new therapies command premium pricing.

Furthermore, strategic collaborations and partnerships between pharmaceutical companies, academic institutions, and healthcare organizations are accelerating the introduction of novel drugs to the market. Regulatory agencies, such as the US FDA and EMA, have also facilitated quicker approval pathways for breakthrough therapies addressing unmet needs in AML. This favorable regulatory environment, combined with the increasing participation of patients in clinical trials, is enabling faster market entry for innovative drugs. The expansion of healthcare infrastructure in emerging economies is also contributing to greater access to AML treatments, further supporting market growth.

In addition to the advancements in acute myeloid leukemia, the field of Acute Lymphoblastic Leukemia Therapeutics is also witnessing significant progress. This type of leukemia, primarily affecting children, has seen improved treatment outcomes due to the development of targeted therapies and immunotherapies. Researchers are focusing on understanding the genetic and molecular basis of the disease to develop more effective treatment protocols. The integration of precision medicine approaches is helping to tailor therapies to individual patient profiles, enhancing efficacy and minimizing side effects. As a result, survival rates for acute lymphoblastic leukemia are improving, offering hope to patients and families affected by this challenging condition.

From a regional perspective, North America continues to dominate the acute myeloid leukemia drugs market, owing to its advanced healthcare infrastructure, high adoption of innovative therapies, and strong presence of key pharmaceutical companies. However, the Asia Pacific region is witnessing the fastest growth, driven by rising healthcare expenditure, increasing awareness about hematological malignancies, and improving access to diagnostics and treatments. Europe also represents a significant market, with robust research activity and government initiatives supporting cancer care. Meanwhile, Latin America and the Middle East & Africa are gradually emerging as promising markets, as local governments invest in cancer care and treatment accessibility.

Acute myeloid leukemia (AML) is a hematological malignancy characterized by clonal expansion of blast cells that exhibit great genetic heterogeneity. In this study, we describe the mutational landscape and its clinico-pathological significance in 26 myeloid neoplasm patients from a South Asian population (Pakistan) by using ultra-deep targeted next-generation DNA sequencing of 54 genes (∼5000×) and its subsequent bioinformatics analysis. The data analysis indicated novel non-silent somatic mutational events previously not reported in AML, including nine non-synonymous and one stop-gain mutations. Notably, two recurrent somatic non-synonymous mutations, i.e., STAG2 (causing p.L526F) and BCORL1 (p.A400V), were observed in three unrelated cases each. The BCOR was found to have three independent non-synonymous somatic mutations in three cases. Further, the SRSF2 with a protein truncating somatic mutation (p.Q88X) was observed for the first time in AML in this study. The prioritization of germline mutations with ClinVar, SIFT, Polyphen2, and Combined Annotation Dependent Depletion (CADD) highlighted 18 predicted deleterious/pathogenic mutations, including two recurrent deleterious mutations, i.e., a novel heterozygous non-synonymous SNV in GATA2 (p.T358P) and a frameshift insertion in NPM1 (p.L258fs), found in two unrelated cases each. The WT1 was observed with three independent potential detrimental germline mutations in three different cases. Collectively, non-silent somatic and/or germline mutations were observed in 23 (88.46%) of the cases (0.92 mutation per case). Furthermore, the pharmGKB database exploration showed a missense SNV rs1042522 in TP53, exhibiting decreased response to anti-cancer drugs, in 19 (73%) of the cases. This genomic profiling of AML provides deep insight into the disease pathophysiology. Identification of pharmacogenomics markers will help to adopt personalized approach for the management of AML patients in Pakistan.

Demographic and clinical data of AML patients and healthy controls of Jordanian Arab descent included in this study.

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APAC Acute Myeloid Leukemia Treatment Market Size 2024-2028

The APAC acute myeloid leukemia (AML) treatment market size is forecast to increase by USD 305.8 million at a CAGR of 13.9% between 2023 and 2028. The market is witnessing significant growth due to the high incidence of this cancer type worldwide. According to the American Cancer Society, AML accounts for approximately one-third of all leukemia diagnoses. A key trend in the market is the adoption of Chimeric Antigen Receptor T-cell (CAR-T) therapy for AML treatment. This innovative approach, which involves engineering a patient's own T-cells to target and destroy cancer cells, has shown promising results in clinical trials. However, challenges remain, particularly in developing nations, where the lack of adequate healthcare infrastructure hampers the widespread adoption of advanced treatment modalities. Despite these challenges, the market is expected to grow steadily due to the increasing demand for effective AML treatments and ongoing research and development efforts.

What will be the Size of the Market During the Forecast Period?

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Acute Myeloid Leukemia (AML), a type of bone marrow cancer characterized by the production of abnormal myeloid cells, is a significant health concern worldwide. Genetic mutations, unhealthy lifestyles, hazardous chemicals, and radiation exposure are known risk factors for this disease. In APAC, the number of AML cases is growing due to population aging and rising awareness about the disease. AML treatment in APAC involves various approaches such as chemotherapy, targeted therapies, and novel therapies. Chemotherapy, which includes induction chemotherapy administered through parenteral or oral routes, is the most common treatment for high-risk AML patients.

Moreover, this therapy aims to destroy the abnormal myeloblasts and stimulate the production of healthy white blood cells. The immune system plays a crucial role in the body's defense against leukemia. However, in some cases, the immune system may not be able to effectively target the abnormal myeloid cells. In such situations, targeted therapies, which use drugs or other substances to specifically target cancer cells, can be effective. Novel therapies, such as gene therapy and stem cell transplantation, are also being explored for the treatment of AML. AML treatment in APAC is provided in hospitals and clinics, including specialized centers. The immune system plays a crucial role in the treatment of AML, with ongoing research focusing on harnessing its potential through cancer immunotherapies. The market for AML treatment is expected to witness significant growth due to the increasing incidence of AML, advancements in technology, and the development of new therapies.

In addition, screening programs are being implemented to increase early detection and improve patient outcomes. The diagnosis of AML involves various tests, including bone marrow aspiration and cytogenetic analysis to identify chromosomal abnormalities. AML can be further classified into two types: myeloblastic leukemia and lymphoblastic leukemia. Myeloblastic leukemia, also known as acute myeloid leukemia with myelodysplasia-related changes, is a subtype of AML characterized by the presence of myelodysplastic syndrome (MDS) features. This subtype is more common in older adults and has a poorer prognosis than other types of AML. In conclusion, the AML treatment market in APAC is witnessing significant growth due to the increasing number of cases and the availability of advanced therapeutic options.

Moreover, chemotherapy, targeted therapies, and novel therapies are the main treatment approaches used in the region. The role of hospitals and clinics, including specialized centers, in providing AML treatment is crucial. Early detection through screening programs and advancements in treatment technologies are key trends driving the growth of the AML treatment market in APAC.

Market Segmentation

The market research report provides comprehensive data (region-wise segment analysis), with forecasts and estimates in 'USD million' for the period 2024-2028, as well as historical data from 2018-2022 for the following segments.

Type

  Chemotherapy
  Stem cell transplantation
  Others


Geography

  APAC

    China
    India
    Japan

By Type Insights

The chemotherapy segment is estimated to witness significant growth during the forecast period.Acute Myeloid Leukemia (AML) is a type of cancer affecting the blood and bone marrow, requiring effective treatment approaches. Chemotherapy is a common treatment method for AML, utilizing drugs such as cytarabine, anthracycline, fludarabine, mitoxantrone, etoposide (VP-16), 6-thioguanine (6-TG), hydroxyurea, corticosteroid drugs, methotrexate (MTX), 6-mercaptopurine (6-MP), azacytidine, decitabine, daunomycin, idarubicin, and mitoxantrone. The chemotherapy segment is expected to

The Cancer Moonshot Biobank is a National Cancer Institute initiative to support current and future investigations into drug resistance and sensitivity and other NCI-sponsored cancer research initiatives, with an aim of improving researchers' understanding of cancer and how to intervene in cancer initiation and progression. During the course of this study, biospecimens (blood and tissue removed during medical procedures) and associated data will be collected longitudinally from at least 1000 patients across at least 10 cancer types, who represent the demographic diversity of the U.S. and receiving standard of care cancer treatment at multiple NCI Community Oncology Research Program (NCORP) sites.

This collection contains de-identified radiology and histopathology imaging procured from subjects in NCI’s Cancer Moonshot Biobank - Acute Myeloid Leukemia Cancer (CMB-AML) cohort. Associated genomic, phenotypic and clinical data will be hosted by The Database of Genotypes and Phenotypes (dbGaP) and other NCI databases. A summary of Cancer Moonshot Biobank imaging efforts can be found on the Cancer Moonshot Biobank Imaging page.

The Acute Myeloid Leukemia (AML) treatment market is a rapidly evolving landscape characterized by significant growth potential. While precise figures for market size and CAGR are unavailable, industry analysis suggests a substantial market driven by an aging population, increasing AML incidence rates, and the continued development of innovative therapies. The market's value likely surpasses several billion dollars, considering the high cost of AML treatments and the significant number of patients requiring long-term care. Key drivers include the rising prevalence of AML, particularly in older adults, and the emergence of targeted therapies offering improved outcomes compared to conventional chemotherapy. These targeted therapies, along with advancements in supportive care and stem cell transplantation, are reshaping the treatment paradigm and pushing market expansion. However, challenges remain, including the high cost of innovative therapies, the development of drug resistance, and the need for improved early detection methods. The competitive landscape includes established pharmaceutical companies like Celgene, Eisai, and Genzyme, alongside smaller biotech firms focused on developing novel targeted treatments. This dynamic interplay of established players and emerging innovators ensures ongoing competition and innovation within the AML treatment market. The forecast period (2025-2033) promises continued growth, driven by ongoing research into novel therapeutic approaches, including immunotherapy, gene therapy, and the development of combination therapies to overcome treatment resistance. Although restraints such as high treatment costs and limited access to advanced therapies in certain regions will persist, the overall market outlook remains positive due to the significant unmet medical needs and the continued investment in research and development. Segment analysis likely reveals a strong focus on targeted therapies and supportive care medications, while regional variations might reflect differences in healthcare infrastructure and access to advanced treatments. The market is anticipated to experience significant growth in the coming years due to the continuous research and development of innovative treatments and an increasing focus on personalized medicine approaches for AML.

Abbreviations: AHA : anti-HLA antibodies;ALL: acute lymphoblastic leukemia; AML: acute myeloid leukemia; ATG:anti-thymocyte globulin; Bu: busulfan; Cy: cyclophosphamide; DSA : Donor-specific anti-HLA antibody; Flu: fludarabine; MDS: myelodisplastic syndrome; Mel: melphalan; MICA : major-histocompatibility-complex class I–related chain A antigens MM: mismatch; MRD: matched related donor; MTX: methotrexate;MUD: matched unrelated donor; VP16:etoposide. AHA of either classes were detected in 31 patients. Class I AHA and class 2 AHA alone were detected in 9 patients, and both in 13 patients, thus 22 (9+13) patients were positive for class I AHA and class II AHA. MICA antibody data was available for only 62 patients out of 70. Data for infused number of CD34+ cells was available for only 64 patients out of 70.

Acute myeloid leukemia (AML) is a malignancy of the myeloid cells due to the clonal and malignant proliferation of blast cells. The etiology of AML is complex and involves environmental and genetic factors. Such genetic aberrations include FLT3, DNMT3, IDH1, IDH2, NAT2, and WT. In this study, we analyzed the relationship between five, not previously studied in any Arab population, single nucleotide polymorphisms (SNPs) and the risk and overall survival of AML in Jordanian patients. The SNPs are NAT2 (rs1799930 and rs1799931), IDH1 (rs121913500), and IDH2 (rs121913502 and rs1057519736). A total number of 30 AML patients and 225 healthy controls were included in this study. Females comprised 50% (n = 15) and 65.3% (n = 147) of patients and controls, respectively. For AML patients (case group) Genomic DNA was extracted from formalin-fixed paraffin-embedded tissues and from peripheral blood samples for the control subjects group. Genotyping of the genetic polymorphisms was conducted using a sequencing protocol. Our study indicates that NAT2 rs1799930 SNP had a statistically significant difference in genotype frequency between cases and controls (p = 0.023) while IDH mutations did not correlate with the risk and survival of AML in the Jordanian population. These results were also similar in the TCGA-LAML cohorts with the notable exception of the rare NAT2 mutation. A larger cohort study is needed to further investigate our results.

Background/Aim: As the knowledgebase of acute myeloid leukemia (AML) has grown, classification systems have moved to incorporate these new findings. Methods: We assessed 32,941 patients with AML whose records are contained in the Surveillance, Epidemiology, and End Results (SEER) database. Results: Half of all patients diagnosed between 2001 and 2013 did not have a World Health Organization (WHO) classification. Acute promyelocytic leukemia and acute panmyelosis with myelofibrosis were associated with the longest leukemia-specific survival (110 and 115 months, respectively), and AML with minimal differentiation and acute megakaryoblastic leukemia with the shortest (30 and 28 months, respectively). For patients in the WHO groups AML not otherwise specified (AML-NOS) and AML with recurrent genetic abnormalities (AML-RGA), the risk of death was greater for older patients and less for married patients. Black patients with any type of AML-NOS also had a higher risk of death. Patients whose case of AML did not receive a WHO classification were older and this group had a higher risk of death when compared to patients with a WHO type of AML-NOS. Conclusion: Our findings highlight the divergent outcomes of patients with AML and the importance of using the WHO classification system and demographic factors to gauge their prognosis.

Acute Myeloid Leukemia Therapeutics Market Outlook

According to our latest research, the global Acute Myeloid Leukemia (AML) therapeutics market size reached USD 2.78 billion in 2024, driven by a robust pipeline of novel therapies and increasing incidence rates worldwide. The market is anticipated to expand at a CAGR of 8.1% during the forecast period, with the market size projected to reach USD 5.47 billion by 2033. This growth is primarily attributed to advancements in targeted therapies, rising awareness about early diagnosis, and improved healthcare infrastructure across emerging economies. The market’s dynamism is further fueled by ongoing research and development activities, which are introducing innovative treatment modalities that are better tolerated and more effective than traditional approaches.

Several key growth factors are propelling the expansion of the Acute Myeloid Leukemia therapeutics market. Foremost among these is the increasing prevalence of AML, particularly among the aging population, as the risk of developing this aggressive hematological malignancy rises significantly with age. Enhanced diagnostic capabilities and greater access to healthcare services have led to earlier detection and, consequently, a higher number of patients being eligible for advanced therapeutics. Additionally, the integration of precision medicine and genomics into clinical practice has enabled the identification of specific genetic mutations, paving the way for personalized treatment regimens that significantly improve patient outcomes. Pharmaceutical companies are increasingly focusing on developing therapies that target these mutations, resulting in a robust pipeline that promises to transform the treatment landscape in the coming years.

Another crucial driver is the evolution of treatment paradigms, particularly the shift from conventional chemotherapy to targeted therapies and immunotherapies. Traditional chemotherapeutic regimens, while effective to some extent, are often associated with significant toxicity and limited long-term efficacy. The emergence of targeted agents and immune checkpoint inhibitors has opened new avenues for patients who are refractory to or ineligible for standard therapies. These novel agents are not only improving survival rates but also offering better quality of life due to their favorable safety profiles. Furthermore, the growing adoption of stem cell transplantation, especially in younger and fit patients, has contributed to improved remission rates and long-term survival, further stimulating market growth.

The market’s momentum is also supported by strategic collaborations between biopharmaceutical companies, academic institutions, and research organizations. These partnerships are accelerating drug discovery and development processes, leading to faster regulatory approvals and market launches of innovative AML therapies. Favorable reimbursement policies and increased government funding for cancer research are additional factors catalyzing market expansion. However, challenges such as high treatment costs, limited access to advanced therapies in low- and middle-income countries, and the risk of relapse continue to pose hurdles. Despite these obstacles, the pipeline of next-generation therapeutics and ongoing clinical trials signal a promising future for the AML therapeutics market.

From a regional perspective, North America currently dominates the global Acute Myeloid Leukemia therapeutics market, accounting for the largest share in 2024, followed by Europe and Asia Pacific. The strong presence of leading pharmaceutical companies, advanced healthcare infrastructure, and high awareness levels contribute to North America’s leadership. Europe’s market is also substantial, driven by supportive government initiatives and a strong focus on research and development. Meanwhile, the Asia Pacific region is witnessing the fastest growth, fueled by rising healthcare expenditure, increasing incidence of AML, and improving access to modern therapies. Latin America and the Middle East & Africa, though smaller in market size, are expected to experience steady growth due to ongoing healthcare reforms and expanded access to innovative treatments.

Treatment Type Analysis

The treatment landscape for Acute Myeloid Leukemia is diverse, encompassing chemotherapy, targeted therapy, immunotherapy, stem cell transplantation, and other emerging modalities. Chemotherapy remains the cornerstone of AML treatment, particularly for newly d

These are peer-reviewed supplementary materials for the article 'Patient characteristics, burden of disease, healthcare resource utilization and costs in acute myeloid leukemia – a retrospective observational study with German claims data' published in the Journal of Comparative Effectiveness Research.Supplementary Table 1: Agents and codes used to categorize incident AML patients into having received intensive chemotherapy (IC) or non-intensive therapy (NIC)Aim: To assess patient characteristics, burden of disease, healthcare resource utilization and costs of acute myeloid leukemia (AML) by treatment intensity in German claims data. Materials & methods: In this retrospective cohort study using claims data from the German sickness fund AOK PLUS, we identified incident AML patients between 2012 and 2022. Incident AML patients were stratified into groups receiving intensive chemotherapy (IC) or nonintensive therapy (NIC). We then conducted descriptive analyses of patient characteristics, disease burden, including blood and platelet transfusions, healthcare resource utilization and costs. Results: We identified 1533 incident AML patients who received treatment (male: 53%; mean age: 67.7 years; median Charlson comorbidity index [CCI]: 5.0), corresponding to an incidence rate of 4.4/100,000. A total of 688 patients (44.9%) were categorized as IC, 845 patients (55.1%) as NIC. Notably, 860 additional patients (male: 48%; 78.0 years; median CCI: 5.0) had no relevant treatment code. NIC patients were older than IC patients (78.0 vs 61.0 years) and had a higher comorbidity burden (median CCI: 6.0 vs 4.0). NIC patients were hospitalized to a lesser extent (81.3% vs 87.9%), had shorter lengths of stay (64.0 vs 103.1 days/patient-year [PY]) and lower hospitalization costs/PY (56,063€/PY vs 110,186€/PY) compared with IC patients. Anemia and thrombocytopenia (NIC: 40.5 and 39.5%, IC: 76.9 and 42.6%) as well as blood and platelet transfusions were common, especially among IC patients (NIC: 93.0 and 74.3%, IC: 99.4 and 98.5%). Conclusion: Compared with IC patients, NIC patients were older, had a higher comorbidity burden and fewer hospitalizations. Combined with the high number of older patients not receiving AML treatment, this points to a lack of adequate treatment options for this patient population. The high rates of blood and platelet transfusions, particularly among IC patients, underscore the high disease burden and emphasize the need for better-tolerated therapies.

Acute myeloid (myelogenous, myelocytic) leukemia (AML) is a rapidly progressing blood cancer with an extremely poor overall prognosis. AML predominantly affects the elderly population and this subgroup of patients has an exceptionally high level of unmet needs. GlobalData estimates the 2014 sales for AML at approximately $342 million across the 7MM covered in this report. The market will increase by almost three-fold over the forecast period, reaching $932 million in 2024 at a CAGR of 10.5%. This growth will be driven by an increase in incident cases of AML as well as the approval and uptake of premium-priced products, such as FLT3 kinase inhibitors (quizartinib and midostaurin), a PI3 kinase inhibitor (volasertib) and new formulations of existing chemotherapies such as Vyxeos (CPX-351), guadecitabine and CC-486. GlobalData expects, by the end of the forecast period, new branded therapies Vyxeos, volasertib and CC-486 will dominate the market along with generic azacitidine. The AML pipeline is weak in terms of efficacy; however, GlobalData expects none of these drugs to have a major impact on the overall AML market. The challenge for new entrants into the AML market is to meet the critically high unmet needs of AML patients and improve overall survival. One of the opportunities for the companies is to work cooperatively to develop therapies that work in combination with chemotherapy backbone. Read More

BackgroundCell metabolic reprogramming is a hallmark of tumor prognosis, and fatty acid metabolism (FAM) plays a crucial role in the tumor microenvironment (TME). However, the relationship between FAM, TME, and prognosis of acute myeloid leukemia (AML) patients remains elusive.MethodsWe extracted the single-cell RNA sequencing (scRNA-Seq) and bulk transcriptome data of AML patients from the TCGA and GEO databases and assessed the relationship between FAM, TME, and AML patient prognosis. We also performed functional enrichment (FE) assay to evaluate the significance of FAM in anti-AML immunosurveillance.ResultsOur scRNA-Seq analysis revealed that the leukemic stem cell (LSC)-enriched population exhibited elevated levels of FAM-related genes. Using these FAM-related genes, we developed a prognostic model that accurately estimated AML patient outcome. FE analysis showed that FAM was strongly related to alterations of TME-based immunosurveillance in AML patients. More importantly, we demonstrated that FAM inhibition via pharmaceutical targeting of PLA2G4A, a highly expressed FAM gene in AML patients with poor prognosis, enhanced the NK cell-mediated immunosurveillance in leukemia cells.ConclusionsLeukemic stem cell (LSC)-enriched population exhibited elevated levels of FAM-related genes. We have successfully established the FAM formula that predicts AML patient prognosis and alterations in the TME-based immunosurveillance. We also found that PLA2G4A was a highly expressed FAM gene in AML patients with poor prognoses. Pharmaceutical targeting of PLA2G4A increased the expression of NKG2DL in leukemia cells in vitro and thus enhanced the NK cell-mediated immunosurveillance.