xml: <clinical_study>
    <!-- This xml conforms to an XML Schema at:
      https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
    <required_header>
      <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
      <link_text>Link to the current ClinicalTrials.gov record.</link_text>
      <url>https://clinicaltrials.gov/ct2/show/NCT06006949</url>
    </required_header>
    <id_info>
      <org_study_id>Lus-2</org_study_id>
      <nct_id>NCT06006949</nct_id>
    </id_info>
    <brief_title>Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory MDS-RS</brief_title>
    <official_title>Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory Myelodysplastic Syndrome With Ring Sideroblasts (MDS-RS): A Prospective Randomized Controlled Study</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Peking Union Medical College Hospital</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Peking Union Medical College Hospital</source>
    <oversight_info>
      <has_dmc>No</has_dmc>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        In a randomized controlled phase II/III clinical trial, 58% of patients with lower-risk MDS&#xD;
        had at least a 50% reduction in red blood cell (RBC) transfusion units every 8 weeks after&#xD;
        roxadustat treatment. In a randomized controlled phase III clinical trial, luspatercept&#xD;
        significantly improved transfusion dependence in erythropoietin-stimulating agents&#xD;
        (ESA)-refractory MDS-RS and improved hemoglobin response and quality of life, compared to&#xD;
        placebo. This study aimed to evaluate the efficacy and safety of roxadustat combined with&#xD;
        luspatercept versus luspatercept monotherapy in the treatment of refractory MDS-RS.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        Myelodysplastic neoplasms (MDS) are heterogeneous clonal disorders of stem cells that result&#xD;
        in peripheral blood cytopenia and ineffective hematopoiesis, with the potential risk of the&#xD;
        development of acute myeloid leukemia (AML). Most patients with myelodysplastic syndromes&#xD;
        with ring sideroblasts (MDS-RS) are stratified into lower-risk groups by the revised&#xD;
        International Prognostic Scoring System (IPSS). At present, the main therapies for MDS-RS are&#xD;
        red blood cell and platelet transfusion, erythropoietin (EPO), androgen, and iron chelation&#xD;
        therapy. Roxadustat can up-regulate transferrin receptors to increase iron absorption,&#xD;
        up-regulate transferrin to promote iron transport, and down-regulate ferritin levels to&#xD;
        indirectly improve iron absorption and transport, promote plasma iron entry into the bone&#xD;
        marrow to generate red blood cells and promote the production of EPO in the physiological&#xD;
        range. Luspatercept generally promotes advanced erythrocyte maturation by inhibiting the&#xD;
        TGF-β/smad2/3 signaling pathway. In a randomized controlled phase II/III clinical trial, 58%&#xD;
        of patients with lower-risk MDS had at least a 50% reduction in red blood cell (RBC)&#xD;
        transfusion units every 8 weeks after roxadustat treatment. In a randomized controlled phase&#xD;
        III clinical trial, luspatercept significantly improved transfusion dependence in&#xD;
        erythropoietin-stimulating agents (ESA)-refractory MDS-RS and improved hemoglobin response&#xD;
        and quality of life, compared to placebo. The aim of this study was to evaluate the efficacy&#xD;
        and safety of roxadustat combined with luspatercept versus luspatercept monotherapy in the&#xD;
        treatment of refractory MDS-RS. If it is proved that the combination of the two drugs is&#xD;
        better than luspatercept monotherapy, it can quickly improve the anemia of refractory MDS-RS&#xD;
        and improve the quality of life.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">August 2023</start_date>
    <completion_date type="Anticipated">August 2025</completion_date>
    <primary_completion_date type="Anticipated">August 2024</primary_completion_date>
    <phase>Phase 4</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>Randomized</allocation>
      <intervention_model>Parallel Assignment</intervention_model>
      <primary_purpose>Treatment</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>overall response rate (ORR)</measure>
      <time_frame>6 month</time_frame>
      <description>Proportion of patients who achieved complete response, partial response and hematological response.</description>
    </primary_outcome>
    <secondary_outcome>
      <measure>transfusion indenpendence (TI)</measure>
      <time_frame>6 month</time_frame>
      <description>Proportion of patients with transfusion dependence over 8 weeks</description>
    </secondary_outcome>
    <secondary_outcome>
      <measure>adverse event rate</measure>
      <time_frame>6 month</time_frame>
      <description>Proportion of patients with adverse events</description>
    </secondary_outcome>
    <number_of_arms>2</number_of_arms>
    <enrollment type="Anticipated">62</enrollment>
    <condition>Myelodysplastic Syndromes</condition>
    <arm_group>
      <arm_group_label>roxadustat and luspatercept</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg.&#xD;
  Roxadustat (150mgqod) was administered for at least 6 months to evaluate efficacy.&#xD;
  Hemoglobin ≥120g/L can be discontinued, and hemoglobin &lt;120g/L can continue to use. Those who are effective will continue to be given the combination therapy until ineffective or intolerant.</description>
    </arm_group>
    <arm_group>
      <arm_group_label>luspatercept</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg.&#xD;
  Luspatercept was given for at least 6 months to evaluate the efficacy. Hemoglobin ≥120g/L can be discontinued, and hemoglobin &lt;120g/L can continue to use. Those who are effective will continue to be given the therapy until it is ineffective or intolerant</description>
    </arm_group>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Roxadustat</intervention_name>
      <description>Roxadustat (150 mgqod)</description>
      <arm_group_label>roxadustat and luspatercept</arm_group_label>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Luspatercept</intervention_name>
      <description>Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg)</description>
      <arm_group_label>luspatercept</arm_group_label>
      <arm_group_label>roxadustat and luspatercept</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            1. Age &gt;18 years old.&#xD;
  &#xD;
            2. Patients with a definite diagnosis of MDS-RS and stratified as lower-risk according to&#xD;
               IPSS-R.&#xD;
  &#xD;
            3. After at least 6 weeks of rhEPO treatment, with hemoglobin&lt;100g/L&#xD;
  &#xD;
            4. Adequate hepatic functions with alanine transaminase (ALT)/aspartate. transaminase&#xD;
               (AST) levels within 2 times of the normal upper limit and total bilirubin levels&#xD;
               within 2 times of the normal upper limit.&#xD;
  &#xD;
            5. ECOG≤2 with an expected life span of more than 6 months&#xD;
  &#xD;
            6. Documented patient consent.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            1. Age &lt;18 years old.&#xD;
  &#xD;
            2. Complicated with active or uncontrolled infections.&#xD;
  &#xD;
            3. Complicated with other malignancies.&#xD;
  &#xD;
            4. Creatinine/transaminase ≥ 2 normal upper limit.&#xD;
  &#xD;
            5. Complicated with myelofibrosis.&#xD;
  &#xD;
            6. Pregnant or lactating women, or men with recent fertility needs&#xD;
  &#xD;
            7. Allergic to luspatercept or excipients&#xD;
  &#xD;
            8. Patients with history of polysorbate 80 allergy&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>18 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_contact>
      <last_name>Bing Han</last_name>
      <phone>13601059938</phone>
      <email>hanbing_li@sina.com.cn</email>
    </overall_contact>
    <location>
      <facility>
        <name>Peking union medical college hospital</name>
        <address>
          <city>Beijing</city>
          <country>China</country>
        </address>
      </facility>
      <contact>
        <last_name>Bing Han, Doctor</last_name>
        <phone>+86-010-69155760</phone>
        <email>hanbingtg123@163.com</email>
      </contact>
    </location>
    <location_countries>
      <country>China</country>
    </location_countries>
    <reference>
      <citation>Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, Bloomfield CD, Cazzola M, Vardiman JW. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016 May 19;127(20):2391-405. doi: 10.1182/blood-2016-03-643544. Epub 2016 Apr 11.</citation>
      <PMID>27069254</PMID>
    </reference>
    <reference>
      <citation>Zheng Q, Yang H, Fu X, Huang Y, Wei R, Wang Y, Liu YN, Liu WJ. The efficacy and safety of roxadustat for anemia in patients with chronic kidney disease: a meta-analysis. Nephrol Dial Transplant. 2021 Aug 27;36(9):1603-1615. doi: 10.1093/ndt/gfaa110.</citation>
      <PMID>33051677</PMID>
    </reference>
    <reference>
      <citation>Gupta N, Wish JB. Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitors: A Potential New Treatment for Anemia in Patients With CKD. Am J Kidney Dis. 2017 Jun;69(6):815-826. doi: 10.1053/j.ajkd.2016.12.011. Epub 2017 Feb 24. Erratum In: Am J Kidney Dis. 2017 Jun;69(6):869.</citation>
      <PMID>28242135</PMID>
    </reference>
    <reference>
      <citation>Crugliano G, Serra R, Ielapi N, Battaglia Y, Coppolino G, Bolignano D, Bracale UM, Pisani A, Faga T, Michael A, Provenzano M, Andreucci M. Hypoxia-Inducible Factor Stabilizers in End Stage Kidney Disease: &quot;Can the Promise Be Kept?&quot;. Int J Mol Sci. 2021 Nov 22;22(22):12590. doi: 10.3390/ijms222212590.</citation>
      <PMID>34830468</PMID>
    </reference>
    <reference>
      <citation>Chen N, Hao C, Liu BC, Lin H, Wang C, Xing C, Liang X, Jiang G, Liu Z, Li X, Zuo L, Luo L, Wang J, Zhao MH, Liu Z, Cai GY, Hao L, Leong R, Wang C, Liu C, Neff T, Szczech L, Yu KP. Roxadustat Treatment for Anemia in Patients Undergoing Long-Term Dialysis. N Engl J Med. 2019 Sep 12;381(11):1011-1022. doi: 10.1056/NEJMoa1901713. Epub 2019 Jul 24.</citation>
      <PMID>31340116</PMID>
    </reference>
    <reference>
      <citation>Henry DH, Glaspy J, Harrup R, Mittelman M, Zhou A, Carraway HE, Bradley C, Saha G, Modelska K, Bartels P, Leong R, Yu KP. Roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndrome: Open-label, dose-selection, lead-in stage of a phase 3 study. Am J Hematol. 2022 Feb 1;97(2):174-184. doi: 10.1002/ajh.26397. Epub 2021 Nov 9.</citation>
      <PMID>34724251</PMID>
    </reference>
    <reference>
      <citation>Kubasch AS, Fenaux P, Platzbecker U. Development of luspatercept to treat ineffective erythropoiesis. Blood Adv. 2021 Mar 9;5(5):1565-1575. doi: 10.1182/bloodadvances.2020002177.</citation>
      <PMID>33687432</PMID>
    </reference>
    <reference>
      <citation>Attie KM, Allison MJ, McClure T, Boyd IE, Wilson DM, Pearsall AE, Sherman ML. A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers. Am J Hematol. 2014 Jul;89(7):766-70. doi: 10.1002/ajh.23732. Epub 2014 Apr 26.</citation>
      <PMID>24715706</PMID>
    </reference>
    <reference>
      <citation>Suragani RN, Cadena SM, Cawley SM, Sako D, Mitchell D, Li R, Davies MV, Alexander MJ, Devine M, Loveday KS, Underwood KW, Grinberg AV, Quisel JD, Chopra R, Pearsall RS, Seehra J, Kumar R. Transforming growth factor-beta superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis. Nat Med. 2014 Apr;20(4):408-14. doi: 10.1038/nm.3512. Epub 2014 Mar 23.</citation>
      <PMID>24658078</PMID>
    </reference>
    <reference>
      <citation>Markham A. Luspatercept: First Approval. Drugs. 2020 Jan;80(1):85-90. doi: 10.1007/s40265-019-01251-5.</citation>
      <PMID>31939073</PMID>
    </reference>
    <reference>
      <citation>Fenaux P, Platzbecker U, Mufti GJ, Garcia-Manero G, Buckstein R, Santini V, Diez-Campelo M, Finelli C, Cazzola M, Ilhan O, Sekeres MA, Falantes JF, Arrizabalaga B, Salvi F, Giai V, Vyas P, Bowen D, Selleslag D, DeZern AE, Jurcic JG, Germing U, Gotze KS, Quesnel B, Beyne-Rauzy O, Cluzeau T, Voso MT, Mazure D, Vellenga E, Greenberg PL, Hellstrom-Lindberg E, Zeidan AM, Ades L, Verma A, Savona MR, Laadem A, Benzohra A, Zhang J, Rampersad A, Dunshee DR, Linde PG, Sherman ML, Komrokji RS, List AF. Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes. N Engl J Med. 2020 Jan 9;382(2):140-151. doi: 10.1056/NEJMoa1908892.</citation>
      <PMID>31914241</PMID>
    </reference>
    <reference>
      <citation>Feld J, Navada SC, Silverman LR. Myelo-deception: Luspatercept &amp; TGF-Beta ligand traps in myeloid diseases &amp; anemia. Leuk Res. 2020 Oct;97:106430. doi: 10.1016/j.leukres.2020.106430. Epub 2020 Jul 30.</citation>
      <PMID>32763582</PMID>
    </reference>
    <reference>
      <citation>Komrokji RS, Platzbecker U, Fenaux P, Zeidan AM, Garcia-Manero G, Mufti GJ, Santini V, Diez-Campelo M, Finelli C, Jurcic JG, Greenberg PL, Sekeres MA, DeZern AE, Savona MR, Shetty JK, Ito R, Zhang G, Ha X, Backstrom JT, Verma A. Luspatercept for myelodysplastic syndromes/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis. Leukemia. 2022 May;36(5):1432-1435. doi: 10.1038/s41375-022-01521-4. Epub 2022 Feb 26. No abstract available.</citation>
      <PMID>35220402</PMID>
    </reference>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 17, 2023</study_first_submitted>
    <study_first_submitted_qc>August 17, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 23, 2023</study_first_posted>
    <last_update_submitted>August 17, 2023</last_update_submitted>
    <last_update_submitted_qc>August 17, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 23, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Principal Investigator</responsible_party_type>
      <investigator_affiliation>Peking Union Medical College Hospital</investigator_affiliation>
      <investigator_full_name>Bing Han</investigator_full_name>
      <investigator_title>Professor</investigator_title>
    </responsible_party>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Preleukemia</mesh_term>
      <mesh_term>Myelodysplastic Syndromes</mesh_term>
      <mesh_term>Syndrome</mesh_term>
    </condition_browse>
    <intervention_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Luspatercept</mesh_term>
    </intervention_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: download date: ClinicalTrials.gov processed this data on September 20, 2023
  link text: Link to the current ClinicalTrials.gov record.
  url: https://clinicaltrials.gov/ct2/show/NCT06006949
  org study id: Lus-2
  nct id: NCT06006949
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  In a randomized controlled phase II/III clinical trial, 58% of patients with lower-risk MDS
        had at least a 50% reduction in red blood cell (RBC) transfusion units every 8 weeks after
        roxadustat treatment. In a randomized controlled phase III clinical trial, luspatercept
        significantly improved transfusion dependence in erythropoietin-stimulating agents
        (ESA)-refractory MDS-RS and improved hemoglobin response and quality of life, compared to
        placebo. This study aimed to evaluate the efficacy and safety of roxadustat combined with
        luspatercept versus luspatercept monotherapy in the treatment of refractory MDS-RS.
  Myelodysplastic neoplasms (MDS) are heterogeneous clonal disorders of stem cells that result
        in peripheral blood cytopenia and ineffective hematopoiesis, with the potential risk of the
        development of acute myeloid leukemia (AML). Most patients with myelodysplastic syndromes
        with ring sideroblasts (MDS-RS) are stratified into lower-risk groups by the revised
        International Prognostic Scoring System (IPSS). At present, the main therapies for MDS-RS are
        red blood cell and platelet transfusion, erythropoietin (EPO), androgen, and iron chelation
        therapy. Roxadustat can up-regulate transferrin receptors to increase iron absorption,
        up-regulate transferrin to promote iron transport, and down-regulate ferritin levels to
        indirectly improve iron absorption and transport, promote plasma iron entry into the bone
        marrow to generate red blood cells and promote the production of EPO in the physiological
        range. Luspatercept generally promotes advanced erythrocyte maturation by inhibiting the
        TGF-β/smad2/3 signaling pathway. In a randomized controlled phase II/III clinical trial, 58%
        of patients with lower-risk MDS had at least a 50% reduction in red blood cell (RBC)
        transfusion units every 8 weeks after roxadustat treatment. In a randomized controlled phase
        III clinical trial, luspatercept significantly improved transfusion dependence in
        erythropoietin-stimulating agents (ESA)-refractory MDS-RS and improved hemoglobin response
        and quality of life, compared to placebo. The aim of this study was to evaluate the efficacy
        and safety of roxadustat combined with luspatercept versus luspatercept monotherapy in the
        treatment of refractory MDS-RS. If it is proved that the combination of the two drugs is
        better than luspatercept monotherapy, it can quickly improve the anemia of refractory MDS-RS
        and improve the quality of life.
  allocation: Randomized
  intervention model: Parallel Assignment
  primary purpose: Treatment
  masking: None (Open Label)
  measure: overall response rate (ORR)
  time frame: 6 month
  description: Proportion of patients who achieved complete response, partial response and hematological response.
  measure: transfusion indenpendence (TI)
  time frame: 6 month
  description: Proportion of patients with transfusion dependence over 8 weeks
  measure: adverse event rate
  time frame: 6 month
  description: Proportion of patients with adverse events
  arm group label: roxadustat and luspatercept
  arm group type: Experimental
  description: Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg.
  Roxadustat (150mgqod) was administered for at least 6 months to evaluate efficacy.
  Hemoglobin ≥120g/L can be discontinued, and hemoglobin <120g/L can continue to use. Those who are effective will continue to be given the combination therapy until ineffective or intolerant.
  arm group label: luspatercept
  arm group type: Experimental
  description: Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg.
  Luspatercept was given for at least 6 months to evaluate the efficacy. Hemoglobin ≥120g/L can be discontinued, and hemoglobin <120g/L can continue to use. Those who are effective will continue to be given the therapy until it is ineffective or intolerant
  intervention type: Drug
  intervention name: Roxadustat
  description: Roxadustat (150 mgqod)
  arm group label: roxadustat and luspatercept
  intervention type: Drug
  intervention name: Luspatercept
  description: Luspatercept (1.0 mg/kg, subcutaneously injection every 3 weeks, adjusted according to blood pattern, up to 1.75mg/kg)
  arm group label: luspatercept
  arm group label: roxadustat and luspatercept
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: N/A
  healthy volunteers: No
  last name: Bing Han
  phone: 13601059938
  email: hanbing_li@sina.com.cn
  facility: 
  contact: 
  country: China
  citation: Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, Bloomfield CD, Cazzola M, Vardiman JW. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016 May 19;127(20):2391-405. doi: 10.1182/blood-2016-03-643544. Epub 2016 Apr 11.
  PMID: 27069254
  citation: Zheng Q, Yang H, Fu X, Huang Y, Wei R, Wang Y, Liu YN, Liu WJ. The efficacy and safety of roxadustat for anemia in patients with chronic kidney disease: a meta-analysis. Nephrol Dial Transplant. 2021 Aug 27;36(9):1603-1615. doi: 10.1093/ndt/gfaa110.
  PMID: 33051677
  citation: Gupta N, Wish JB. Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitors: A Potential New Treatment for Anemia in Patients With CKD. Am J Kidney Dis. 2017 Jun;69(6):815-826. doi: 10.1053/j.ajkd.2016.12.011. Epub 2017 Feb 24. Erratum In: Am J Kidney Dis. 2017 Jun;69(6):869.
  PMID: 28242135
  citation: Crugliano G, Serra R, Ielapi N, Battaglia Y, Coppolino G, Bolignano D, Bracale UM, Pisani A, Faga T, Michael A, Provenzano M, Andreucci M. Hypoxia-Inducible Factor Stabilizers in End Stage Kidney Disease: "Can the Promise Be Kept?". Int J Mol Sci. 2021 Nov 22;22(22):12590. doi: 10.3390/ijms222212590.
  PMID: 34830468
  citation: Chen N, Hao C, Liu BC, Lin H, Wang C, Xing C, Liang X, Jiang G, Liu Z, Li X, Zuo L, Luo L, Wang J, Zhao MH, Liu Z, Cai GY, Hao L, Leong R, Wang C, Liu C, Neff T, Szczech L, Yu KP. Roxadustat Treatment for Anemia in Patients Undergoing Long-Term Dialysis. N Engl J Med. 2019 Sep 12;381(11):1011-1022. doi: 10.1056/NEJMoa1901713. Epub 2019 Jul 24.
  PMID: 31340116
  citation: Henry DH, Glaspy J, Harrup R, Mittelman M, Zhou A, Carraway HE, Bradley C, Saha G, Modelska K, Bartels P, Leong R, Yu KP. Roxadustat for the treatment of anemia in patients with lower-risk myelodysplastic syndrome: Open-label, dose-selection, lead-in stage of a phase 3 study. Am J Hematol. 2022 Feb 1;97(2):174-184. doi: 10.1002/ajh.26397. Epub 2021 Nov 9.
  PMID: 34724251
  citation: Kubasch AS, Fenaux P, Platzbecker U. Development of luspatercept to treat ineffective erythropoiesis. Blood Adv. 2021 Mar 9;5(5):1565-1575. doi: 10.1182/bloodadvances.2020002177.
  PMID: 33687432
  citation: Attie KM, Allison MJ, McClure T, Boyd IE, Wilson DM, Pearsall AE, Sherman ML. A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers. Am J Hematol. 2014 Jul;89(7):766-70. doi: 10.1002/ajh.23732. Epub 2014 Apr 26.
  PMID: 24715706
  citation: Suragani RN, Cadena SM, Cawley SM, Sako D, Mitchell D, Li R, Davies MV, Alexander MJ, Devine M, Loveday KS, Underwood KW, Grinberg AV, Quisel JD, Chopra R, Pearsall RS, Seehra J, Kumar R. Transforming growth factor-beta superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis. Nat Med. 2014 Apr;20(4):408-14. doi: 10.1038/nm.3512. Epub 2014 Mar 23.
  PMID: 24658078
  citation: Markham A. Luspatercept: First Approval. Drugs. 2020 Jan;80(1):85-90. doi: 10.1007/s40265-019-01251-5.
  PMID: 31939073
  citation: Fenaux P, Platzbecker U, Mufti GJ, Garcia-Manero G, Buckstein R, Santini V, Diez-Campelo M, Finelli C, Cazzola M, Ilhan O, Sekeres MA, Falantes JF, Arrizabalaga B, Salvi F, Giai V, Vyas P, Bowen D, Selleslag D, DeZern AE, Jurcic JG, Germing U, Gotze KS, Quesnel B, Beyne-Rauzy O, Cluzeau T, Voso MT, Mazure D, Vellenga E, Greenberg PL, Hellstrom-Lindberg E, Zeidan AM, Ades L, Verma A, Savona MR, Laadem A, Benzohra A, Zhang J, Rampersad A, Dunshee DR, Linde PG, Sherman ML, Komrokji RS, List AF. Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes. N Engl J Med. 2020 Jan 9;382(2):140-151. doi: 10.1056/NEJMoa1908892.
  PMID: 31914241
  citation: Feld J, Navada SC, Silverman LR. Myelo-deception: Luspatercept & TGF-Beta ligand traps in myeloid diseases & anemia. Leuk Res. 2020 Oct;97:106430. doi: 10.1016/j.leukres.2020.106430. Epub 2020 Jul 30.
  PMID: 32763582
  citation: Komrokji RS, Platzbecker U, Fenaux P, Zeidan AM, Garcia-Manero G, Mufti GJ, Santini V, Diez-Campelo M, Finelli C, Jurcic JG, Greenberg PL, Sekeres MA, DeZern AE, Savona MR, Shetty JK, Ito R, Zhang G, Ha X, Backstrom JT, Verma A. Luspatercept for myelodysplastic syndromes/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis. Leukemia. 2022 May;36(5):1432-1435. doi: 10.1038/s41375-022-01521-4. Epub 2022 Feb 26. No abstract available.
  PMID: 35220402
  responsible party type: Principal Investigator
  investigator affiliation: Peking Union Medical College Hospital
  investigator full name: Bing Han
  investigator title: Professor
  mesh term: Preleukemia
  mesh term: Myelodysplastic Syndromes
  mesh term: Syndrome
  mesh term: Luspatercept
  sharing ipd: No