hydroxyurea
/ Generic mfg.
- LARVOL DELTA
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September 21, 2026
Hydroxyurea-coordinated copper-based metal-organic framework nanoreactor for glucose-gated nitric oxide release and infected wound treatment.
(PubMed, J Colloid Interface Sci)
- "GOx@CuPHcs was also associated with macrophage phenotype remodeling, enhanced fibroblast migration, and accelerated wound healing in a diabetic murine model with negligible systemic toxicity. This work demonstrates that coordination interactions can improve the stability of hydrolytically labile therapeutic molecules while enabling their integration into stimulus-responsive nanoreactors, highlighting the potential of coordination-based strategies for developing advanced therapeutic systems."
Journal • Diabetes • Genetic Disorders • Hematological Disorders • Infectious Disease • Sickle Cell Disease
November 03, 2023
Firstline Treatment with Ruxolitinib Versus Best Available Therapy in Patients with Polycythemia Vera: Pre-Specified Interim Analysis of the Randomized Phase 2b Ruxobeat Clinical Trial of the German Study Group for Myeloproliferative Neoplasms (GSG-MPN)
(ASH 2023)
- P2 | "Cytoreductive treatment with hydroxyurea (HU) or ropeginterferon-alpha is approved in EU for the treatment of high-risk patients (pts) with PV. In this interim analysis, first-line treatment with ruxolitinib for 6 months in high-risk pts with PV led to clinically meaningful improvements in overall response, hemoglobin and hematocrit, phlebotomy rates, splenomegaly, and patient-reported pruritus and fatigue severity, while BAT only improved platelet counts, WBC, hematocrit, and phlebotomy rates, without having an impact on symptoms."
Clinical • P2b data • Dermatology • Fatigue • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Pruritus
November 04, 2022
Ruxolitinib Versus Best Available Therapy in Patients with Essential Thrombocythemia: Pre-Specified Interim Analysis of the Randomized Phase 2b Ruxobeat Clinical Trial of the German Study Group for Myeloproliferative Neoplasms (GSG-MPN)
(ASH 2022)
- P2 | "Cytoreductive treatment with hydroxyurea (HU) or anagrelide is approved for the treatment of patients (pts) with ET. In this pre-specified interim analysis, treatment with ruxolitinib was not superior over BAT to induce complete response (using strict criteria for symptoms) in ET pts that were either untreated or not intolerant/resistant to prior therapy. However, RUX was more effective in reducing ET-associated spleen size and symptoms, such as headache and concentration problems. The RuxoBEAT trial is ongoing."
Clinical • P2b data • Cardiovascular • Dermatology • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Pain • Polycythemia Vera • Pruritus • Thrombocytosis
September 10, 2026
Beyond the Bone Marrow: A Case Report of Extramedullary Hematopoiesis in Advanced Primary Myelofibrosis.
(PubMed, Cureus)
- "The patient received hydroxyurea, ruxolitinib, and palliative hepatosplenic radiotherapy, achieving transient clinical and biochemical improvement. However, he subsequently developed radiation-induced bone marrow aplasia and died from progressive disease. This case underscores the aggressive clinical course of advanced PMF when continuous disease-modifying therapy is interrupted and highlights the importance of timely access to targeted treatment and a multidisciplinary approach for managing extensive EMH and its life-threatening complications."
Journal • Acute Myelogenous Leukemia • Aplastic Anemia • Cardiovascular • Cholestasis • Chronic Eosinophilic Leukemia • Fibrosis • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Hepatology • Hypertension • Immunology • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Portal Hypertension
August 18, 2026
FLAsH-IV-AML: Addition of Hydroxyurea to Fludarabine, Cytarabine, Idarubicin and Venetoclax Salvage Therapy for Adults With Relapsed or Refractory Acute Myeloid Leukemia
(clinicaltrials.gov)
- P1/2 | N=26 | Not yet recruiting | Sponsor: Christer C Nilsson
New P1/2 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology
November 06, 2024
IO-202, a Novel Anti-LILRB4 Antibody, with Azacitidine for Hypomethylating Agent-Naive Chronic Myelomonocytic Leukemia: Phase 1b Expansion Cohort Results
(ASH 2024)
- P1 | "Five patients had prior therapies including 4 with hydroxyurea and 1 with fedratinib. Translational data suggest LILRB4 expression as a biomarker for response to therapy, supporting the mechanism of action for IO-202. In light of the paucity of effective therapies for CMML, these data support a future pivotal study of IO-202 + AZA in HMA-naïve CMML."
P1 data • Anemia • Chronic Myelomonocytic Leukemia • Dermatology • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Pruritus • ASXL1 • LILRB4 • RUNX1 • SRSF2 • TET2
September 01, 2026
Real-World Patient Characteristics, Treatment Patterns, and Blood Counts of Patients With Essential Thrombocythemia Treated With Ropeginterferon Alfa-2b in the US Community Oncology Setting
(SOHO 2026)
- "In patients with ≥1 prior cytoreductive therapy (n = 49), the most common 1L was hydroxyurea (92%), and median time from ET diagnosis to ropeginterferon initiation was 13 months. In patients with ≥2 prior cytoreductive therapies (n = 21), next cytoreductive therapies after 1L included anagrelide (43%), peginterferon (24%), and ruxolitinib (19%)... This analysis provides preliminary insights into real-world use and outcomes of ropeginterferon in patients with ET. Ropeginterferon was largely initiated after 1L but also as 1L for younger patients. Platelets and response rates trended towards optimal levels regardless of cytoreductive therapy history."
Clinical • Real-world • Real-world evidence • Essential Thrombocythemia • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera
September 01, 2026
Chronic Neutrophilic Leukemia With the CSF3R T618I Mutation: Diagnostic and Therapeutic Challenges in a BCR::ABL1-Negative Myeloproliferative Neoplasm
(SOHO 2026)
- "The patient was initially started on hydroxyurea, but it was halted due to worsening anemia. Maxson et al reported considerable clinical improvement with ruxolitinib in a patient harboring this mutation.1 This case highlights the importance of comprehensive molecular testing in assessing persistent neutrophilia and emphasizes the diagnostic and therapeutic significance of the CSF3R T618I mutation. Ongoing clinical trials and consensus-based guidelines are needed to advance the management strategies for this rare hematological cancer."
Chronic Neutrophilic Leukemia • Hematological Malignancies • Leukemia • Myeloproliferative Neoplasm • Oncology • ABL1 • BCR • CALR • CSF3R • MPL
September 01, 2026
A Rare Case of ZBTB16::RARA Fusion Acute Myeloid Leukemia Mimicking Acute Promyelocytic Leukemia: Diagnostic Challenges and Successful CLIA + Venetoclax Therapy
(SOHO 2026)
- "The zinc finger and BTB domain-containing 16 (ZBTB16):: retinoic acid receptor alpha (RARA) fusion, also known as promyelocytic leukemia zinc finger (PLZF)::RARA, is a rare variant RARA fusion rearrangement (<1% of cases) that phenotypically mimics acute promyelocytic leukemia (APL) but is resistant to alltrans retinoic acid (ATRA) and arsenic trioxide (ATO) due to the PLZF repressive domain...The patient received induction cladribine, idarubicin, cytarabine (CLIA) plus venetoclax...Supportive care included hydroxyurea, transfusions, and prophylaxis...Venetoclax-based regimens represent a promising approach in this rare entity. Long-term follow-up and potential consolidation with transplant are planned."
Clinical • IO biomarker • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • Wilms Tumor • CD2 • CD34 • CD38 • KIT • NCAM1 • RARA • SMARCB1 • SMARCD3 • WT1 • ZBTB16
September 01, 2026
Real-World Phlebotomy Burden and Treatment Gaps in United States Patients With Polycythemia Vera: A Chart Review Across Physician Practice Settings
(SOHO 2026)
- "At the time of management change from phlebotomy, 47% were receiving concurrent CRT, whereas 45% initiated a new CRT thereafter (most commonly hydroxyurea [42%], ruxolitinib [36%], and ropeginterferon alfa-2b [21%]), and 15% did not receive CRT. Phlebotomy remains widely used in PV care but is associated with substantial procedural burden and frequent management changes. Reporting of inadequate hematocrit control as a driver of management change, treatment-related ID, fatigue, and continued need for management changes despite concurrent CRT collectively point to an urgent need for more effective, better-tolerated treatments."
Clinical • Real-world • Real-world evidence • Review • Oncology • Polycythemia Vera
September 01, 2026
Ruxolitinib-Induced Complete Molecular Remission in Polycythemia Vera
(SOHO 2026)
- "Ruxolitinib (RUX) is a JAK inhibitor approved for treating patients with PV who are nonresponsive to or intolerant of hydroxyurea (HU) and who have demonstrated improved hematocrit control, symptom burden, and reduced variant allele fraction (VAF) vs best available therapy. This patient's hematologic, molecular, and histopathological response to RUX suggests the potential of JAK2 inhibitor-targeted clearance of MPN HSPCs, yet it remains unproven that malignant HSPC eradication is reliably achieved with JAK inhibitors and if this is necessary to achieve cure. A retrospective analysis of patients with PV treated with RUX who achieved deep molecular response might identify determinants of RUX molecular response and potential for cure. BCR::ABL1: breakpoint cluster region–Abelson oncogene homolog 1 fusion gene, CMR: complete molecular remission, EFS: event-free survival, HSPCs: hematopoietic stem and progenitor cells, JAK2: Janus kinase 2, JAK-STAT: Janus kinase/signal..."
Clinical • Essential Thrombocythemia • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • ABL1 • BCR
September 10, 2026
Restricted Anthracycline Strategy in Pediatric Acute Promyelocytic Leukemia - A Single Centre Experience.
(PubMed, Indian J Hematol Blood Transfus)
- "An ATO-ATRA (Arsenic trioxide-All trans retinoic acid) based treatment protocol was employed, but with a unique strategy that restricts anthracycline use to progressive leucocytosis, irrespective of risk stratification. While three were lost to follow-up thereafter, the remaining 9 (75%) completed consolidation and are in long-term molecular remission. Hydroxyurea as a cytoreductive agent in APML allows for restricting anthracycline use to progressive leucocytosis without compromising outcomes."
Journal • Acute Promyelocytic Leukemia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Pediatrics
November 04, 2025
Prospera (ABNL-MARRO 002): A randomized phase 2 study of pacritinib vs. hydroxyurea in patients with advanced proliferative chronic myelomonocytic leukemia (CMML)
(ASH 2025)
- P2 | "In the phase 3 DACOTA trial (Itzykson et al., JCO 2022),decitabine modestly delayed leukemic transformation compared to HU but did not improve event-free oroverall survival and was associated with increased early mortality. The primary endpoint is CBR at Week 24, defined by modified IWG MDS/MPNcriteria incorporating hematologic improvement, spleen volume reduction, and symptom response.Secondary endpoints include CBR at any time, duration of response, event-free survival, leukemia-freesurvival, and overall survival.Correlative studies will assess treatment-related changes in clonal dynamics, cytokine signaling, andhematopoietic composition using longitudinal peripheral blood and bone marrow sampling. Single-celltranscriptomic and immunophenotypic profiling will be employed to characterize pacritinib's impact onboth the malignant clone and the surrounding immune microenvironment."
Clinical • Metastases • P2 data • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytopenia • IRAK1
September 01, 2026
Mortality, Thrombotic, Hemorrhagic, and Cardiovascular Outcomes With JAK1/JAK2/IRAK1 Inhibitors Versus Conventional Cytoreductive Therapy in Myeloproliferative Neoplasms: A Real-World Propensity-Matched Analysis
(SOHO 2026)
- "Patients: Adults aged 18–75 years with MPN-spectrum diagnoses (polycythemia vera, essential thrombocythemia, primary myelofibrosis, chronic myeloproliferative disease, CML, MDS; ICD-10 codes D45, D46, D47.1, D47.3, D47.4, D47.Z9, C92.1) on/after January 1, 2010, initiating a JAK1/JAK2/IRAK1 inhibitor (ruxolitinib, fedratinib, pacritinib, momelotinib; n = 4251) or non-JAK cytoreductive agent (hydroxyurea, anagrelide, interferon alfa-2a/2b; n = 14448) within 3 months of diagnosis... JAK1/JAK2/IRAK1 inhibitor therapy was associated with markedly higher 3-year mortality, hospitalization, major bleeding, VTE, and heart failure compared with non-JAK cytoreductive therapy. Because JAK inhibitors are preferentially used in higher-risk MPN phenotypes imperfectly captured by ICD coding, residual confounding by unmeasured disease severity likely contributes. Prospective comparative effectiveness studies with granular severity adjustment are needed."
Clinical • Real-world • Real-world evidence • Chronic Myeloid Leukemia • Essential Thrombocythemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • IRAK1 • JAK1 • JAK2
September 01, 2026
Ischemic Priapism as the Initial Manifestation of Chronic Myeloid Leukemia: Successful Transition to Asciminib After Dasatinib-Induced Severe Pulmonary Hypertension
(SOHO 2026)
- "After initial cytoreduction with hydroxyurea, he was started on dasatinib. This case highlights priapism as a dramatic hyperviscosity-related presentation of CML-CP. Prompt TKI therapy achieved deep molecular response, while timely transition to asciminib maintained excellent disease control after serious dasatinib-related cardiopulmonary toxicity. Vigilant cardiopulmonary monitoring is crucial in patients on second-generation TKIs."
Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • BCR
May 12, 2023
CHARACTERISTICS AND CLINICAL OUTCOMES IN PATIENTS (PTS) WITH POLYCYTHEMIA VERA (PV) RECEIVING RUXOLITINIB (RUX) AFTER HYDROXYUREA (HU): A LONGITUDINAL ANALYSIS FROM REVEAL
(EHA 2023)
- P=N/A | "This longitudinal analysis of REVEAL demonstrated that HU-RUX had more severe disease burden vs HU. In HU- RUX, RUX tx resulted in significant improvements in PV-related symptoms, spleen size reduction, and sustainedblood parameter improvements. This correlated with a reduction of PHL within 6 mo; most pts became PHL-free at 12 mo."
Clinical • Clinical data • Polycythemia Vera
May 12, 2023
TREATMENT COMPARISON OF HYDROXYUREA VS RUXOLITINIB IN ESSENTIAL THROMBOCYTHEMIA (ET): A MATCHED COHORT ANALYSIS
(EHA 2023)
- P=N/A, P2 | "Pts who switched from hydroxyurea to RUX experienced controlled WBC and PLT counts compared with pts who stayed on hydroxyurea. Pts who switched from hydroxyurea to RUX also showed a reduction in splenomegaly over time. These results demonstrate that switching to RUX after being refractory or intolerant to hydroxyurea may improve clinical outcomes of pts with ET."
Essential Thrombocythemia • Fibrosis • Hematological Disorders • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Thrombocytosis
August 08, 2025
[177Lu]Lu-DOTATATE for Recurrent Meningioma (LUMEN-1, EORTC-2334-BTG): Study Protocol for a Randomized Phase II Trial.
(PubMed, J Nucl Med)
- P2 | "In total, 136 patients will be randomized in a 2:1 ratio to [177Lu]Lu-DOTATATE (≤4 doses of 7.4 GBq given every 4 wk) or local standard of care (hydroxyurea, bevacizumab, sunitinib, octreotide, everolimus, or observation). The trial protocol includes a comprehensive exploratory translational research program with dosimetry and imaging-based and tissue-based investigations. LUMEN-1 was activated in March 2025 and will enroll patients in 35 sites in 10 countries across Europe, with primary endpoint collection planned after 2 y and study completion after 5 y. To our knowledge, EORTC-2334-BTG (LUMEN-1, NCT06326190) is the first prospective randomized trial investigating the efficacy of [177Lu]Lu-DOTATATE in patients with recurrent meningioma."
Journal • P2 data • Brain Cancer • Meningioma • Oncology • Solid Tumor • SSTR • SSTR2
September 06, 2026
Refractory Hypereosinophilic Syndrome With Clonal T-Cell Receptor Rearrangement Treated With JAK Inhibition Following Incomplete Response to IL-5 Blockade.
(PubMed, Case Rep Hematol)
- "Despite prior hydroxyurea therapy, prolonged treatment with mepolizumab, and repeated corticosteroid courses, the patient developed recurrent disease with an absolute eosinophil count of 2760/μL. Ruxolitinib was initiated while IL-5 inhibition was continued...This case highlights the potential role of JAK inhibition as adjunctive therapy in refractory HES with clonal T-cell receptor rearrangement. Further studies are needed to define the safety, durability, and optimal integration of JAK inhibition in patients with refractory eosinophilic disorders."
Journal • Eosinophilia • Hematological Disorders • Hematological Malignancies • Hypereosinophilic Syndrome • Immunology • Oncology • IL5
September 13, 2026
The Impact of Common Medications on Male Fertility: An Updated Systematic Review.
(PubMed, Andrology)
- "In this updated review, evidence-based recommendations for clinical management and for possible fertility preservation or contraception in men of reproductive age receiving common acute or chronic medical treatment are provided, with the goal of making these data more readily available to medical staff."
Journal • Review • Infertility
May 28, 2026
Improving public cancer care by implementing precision medicine in Norway
(clinicaltrialsregister.eu)
- P1/2 | N=1000 | Recruiting | Sponsor: Oslo University Hospital HF | N=6000 ➔ 1000
Enrollment change • Oncology
September 01, 2026
Disparate Outcomes and Treatment Patterns in Polycythemia Vera Across an Integrated Academic-Community Network
(SOHO 2026)
- "Ruxolitinib or interferon use beyond first-line hydroxyurea was lower in NAC vs CCC patients with high-risk PV (29.5% vs 50%, P < 0.01). Within an integrated cancer network, PV treatment patterns and outcomes differed by care site and provider specialty. Compared with CCC or specialist care, community-managed patients had higher incident TEs and worse survival, calling for collaborations to mitigate disparities."
Acute Myelogenous Leukemia • Chronic Eosinophilic Leukemia • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera
December 24, 2022
Efficacy and safety of ruxolitinib in patients with newly-diagnosed polycythemia vera: futility analysis of the RuxoBEAT clinical trial of the GSG-MPN study group.
(PubMed, Ann Hematol)
- P2 | "Ruxolitinib (RUX) is approved for second-line therapy in high-risk PV pts with hydroxyurea intolerance or resistance. One hundred nine adverse events (AEs) occurred in 24/28 patients (all grade 1 to 3), and no pt permanently discontinued treatment because of AEs. Thus, treatment with ruxolitinib in untreated PV pts is feasible, well-tolerated, and efficient regarding the above-mentioned endpoints."
Journal • Cardiovascular • Dermatology • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Pruritus
November 03, 2023
Olverembatinib (HQP1351) Demonstrates Efficacy Vs. Best Available Therapy (BAT) in Patients (Pts) with Tyrosine Kinase Inhibitor (TKI)-Resistant Chronic Myeloid Leukemia Chronic-Phase (CML-CP) in a Registrational Randomized Phase 2 Study
(ASH 2023)
- P2 | "Introduction This was a multicenter, randomized, registrational phase 2 study to assess the efficacy and safety of olverembatinib compared with BAT in pts with CML-CP who were resistant and/or intolerant to 3 TKIs (imatinib [I], dasatinib [D], nilotinib [N]) in China...Pts were randomized 2:1 to investigational olverembatinib (40 mg QOD) or the BAT arm, which could be one of the following per investigator choice: TKIs (I, D, or N), interferon (IFN), hydroxyurea (HU), and homoharringtonine (HHT)...Olverembatinib was observed to be better tolerated and more effective than BAT in treating these pts. Internal study (CT.gov) numbers: HQP1351CC203 (NCT04126681)."
Clinical • P2 data • Anemia • Cardiovascular • Chronic Myeloid Leukemia • CNS Disorders • Congestive Heart Failure • Coronary Artery Disease • Dyslipidemia • Heart Failure • Hematological Disorders • Hematological Malignancies • Hypertriglyceridemia • Leukemia • Leukopenia • Myocardial Infarction • Neutropenia • Oncology • Thrombocytopenia • ABL1
September 01, 2026
Hematocrit Control, Symptom Burden, and Time Toxicity Among Phlebotomy-Treated Patients With Polycythemia Vera in the United States
(SOHO 2026)
- "At survey, PHL+cyto patients were receiving hydroxyurea (64%), ruxolitinib (22%), ropeginterferon alfa-2b (10%), or hydroxyurea plus ruxolitinib combination (4%). In this real-world US cohort, hematocrit control was poor, and symptom burden persisted among phlebotomy-treated patients, including those receiving cytoreductive therapy. Patient-reported mild fatigue and time toxicity indicated a multifactorial burden for phlebotomy-treated patients, with patients losing 3–4 hours of their day per phlebotomy. These findings highlight a clear unmet need for therapies that improve hematocrit and symptom control while reducing time burden."
Clinical • Oncology • Polycythemia Vera
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