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| ID | Type | Description | Link |
|---|---|---|---|
| HEM0056 | Other Identifier | Stanford | |
| NCI-2022-02837 | Registry Identifier | National Cancer Institute: Clinical Trials Reporting Program |
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| Name | Class |
|---|---|
| Celgene Corporation | INDUSTRY |
| Bristol-Myers Squibb | INDUSTRY |
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This is a phase 1b/2, open-label, single arm study to evaluate if enasidenib is safe and effective in improving anemia and decreasing transfusion needs in subjects diagnosed with lower risk myelodysplastic syndrome (MDS) or nonproliferative chronic myelomonocytic leukemia (CMML) without a mutation in isocitrate dehydrogenase type 2 (IDH2 wildtype). Other objectives include assessment of improvements in platelet production and characterization of the mechanism of action of enasidenib in enhancing endogenous erythropoiesis.
Primary Objective(s)- To determine the efficacy (response rate) of enasidenib in improving anemia and decreasing RBC transfusion dependence.
Secondary Objective(s)- To determine the tolerability, safety and durability of the erythroid response and identify laboratory parameters as clinical markers of response.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Enasidenib mesylat | Experimental | Participants will self administer the enasidenib orally everyday. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Enasidenib mesylat dose escalation | Drug | Subjects will participate dose escalation with a starting dose of 100 mg. Enasidenib will be self administered orally and daily. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Response: Hematological Improvement - Erythroid (HI-E) | Clinical response was assessed as the number of participants achieving a hematological improvement - erythroid (HI-E). Participants were characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows.
| 16 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Related Adverse Events | Toxicity was assessed as the number of related non-serious adverse events and related serious adverse events (SAEs) reported by dose level (Cohort A or Cohort B) for the 12-cycle treatment period plus follow-up. | 12 months |
| Time to Hematological Improvement - Erythroid (HI-E) |
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Inclusion Criteria:
Documented diagnosis of
No disease-modifying therapy (HMA, hydrea) within 2 months of starting study
Age ≥ 18 years of age
ECOG ≤ 3
Negative for IDH2 mutation by NGS or multiplex PCR (SNaPshot)
Has symptomatic anemia defined as hemoglobin < 10.5 g/dL with any of the following.
Stated willingness to comply with all study procedures and availability for the duration of the study
Ability to take oral medication and be willing to adhere to the medication regimen.
Females of reproductive potential need to either commit to true abstinence from heterosexual contact or agree to use, and be able to comply with highly effective contraception without interruption, 28 days prior to starting enasidenib, during the study therapy, and for 30 days after last dose of enasidenib
For males of reproductive potential: agreement to use of condoms
Adequate organ function defined as:
Ability to understand and the willingness to sign the IRB approved informed consent document.
Women of childbearing potential must have negative urine or serum pregnancy test
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Tian Yi Zhang, MD | Stanford University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford Cancer Institute | Palo Alto | California | 94305 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Enasidenib Mesylat 100mg | Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily. |
| FG001 | Enasidenib Mesylat 200mg | Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Enasidenib Mesylat 100mg | Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily. |
| BG001 | Enasidenib Mesylat 200mg |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Clinical Response: Hematological Improvement - Erythroid (HI-E) | Clinical response was assessed as the number of participants achieving a hematological improvement - erythroid (HI-E). Participants were characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows.
| Posted | Number | participants | 16 weeks |
|
Informed consent through 90 days after last dose, an average of 9 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Enasidenib Mesylat 100mg | Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 100 mg once daily. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Tian Yi Zhang | Stanford University | 650-497-6259 | tzhang8@stanford.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 23, 2024 | Dec 4, 2025 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D007938 | Leukemia |
| D007951 | Leukemia, Myeloid |
| D007948 | Leukemia, Monocytic, Acute |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C000605269 | enasidenib |
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|
Time to hematological improvement - erythroid (HI-E) was assessed as the time from first dose of enasidenib to the first observed hemoglobin response. Participants will be characterized and stratified as non-transfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows.
|
| 16 weeks |
| Duration of Hematological Improvement - Erythroid (HI-E) | Duration of Hematological Improvement - Erythroid (HI-E) will be assessed as the time from recorded response to loss of response. Participants will be characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows. NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements LTB = 0 units of RBC transfusions HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions The outcome will be reported as the number of participants that achieve the response, a number without dispersion. | 16 weeks |
| Clinical Response: Hematological Improvement - Platelets (HI-P) | Clinical response for platelets was assessed as the number of participants achieving a hematological improvement - platelets (HI-P). Participants will be characterized and stratified as platelets < or ≥ 20 x 10^9/L, with response defined as follows.
| 8 weeks |
| Clinical Response: Hematological Improvement - Neutrophils (HI-N) | Clinical response for neutrophils was assessed as the number of participants achieving a hematological improvement - neutrophils (HI-N). Response was defined as an absolute increase in neutrophils > 0.5 × 10^9/L that was also an increase of ≥ 100%. | 8 weeks |
| Red Blood Cell (RBC) Transfusion Independence (RBC TI) | Clinical response for red blood cells was assessed as the number of participants who were transfusion dependent that achieve red blood cell (RBC) transfusion independence (RBC TI) for for 8 weeks or longer. | 12 months |
Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily.
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG001 | Enasidenib Mesylat 200mg | Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily. |
|
|
| Secondary | Related Adverse Events | Toxicity was assessed as the number of related non-serious adverse events and related serious adverse events (SAEs) reported by dose level (Cohort A or Cohort B) for the 12-cycle treatment period plus follow-up. | Posted | Number | Adverse Events | 12 months |
|
|
|
| Secondary | Time to Hematological Improvement - Erythroid (HI-E) | Time to hematological improvement - erythroid (HI-E) was assessed as the time from first dose of enasidenib to the first observed hemoglobin response. Participants will be characterized and stratified as non-transfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows.
| No participants achieved Hematological Improvement - Erythroid (HI-E), and therefore no results could be calculated. | Posted | 16 weeks |
|
|
| Secondary | Duration of Hematological Improvement - Erythroid (HI-E) | Duration of Hematological Improvement - Erythroid (HI-E) will be assessed as the time from recorded response to loss of response. Participants will be characterized and stratified as nontransfused (NTD), low-transfusion burden (LTB) and high-transfusion burden (HTB), with response defined as follows. NTD = greater than or equal to 2 consecutive Hb measurements, greater than or equal to 1.5 g/dL for a period of minimum 8 week in an observation period of 16 to 24 week compared to the lowest mean of 2 Hb measurements LTB = 0 units of RBC transfusions HTB = greater than or equal to 4 unit or greater than or equal to 50% reduction in RBC transfusions The outcome will be reported as the number of participants that achieve the response, a number without dispersion. | No participants achieved Hematological Improvement - Erythroid (HI-E), and therefore no results could be calculated. | Posted | 16 weeks |
|
|
| Secondary | Clinical Response: Hematological Improvement - Platelets (HI-P) | Clinical response for platelets was assessed as the number of participants achieving a hematological improvement - platelets (HI-P). Participants will be characterized and stratified as platelets < or ≥ 20 x 10^9/L, with response defined as follows.
| Posted | Count of Participants | Participants | 8 weeks |
|
|
|
| Secondary | Clinical Response: Hematological Improvement - Neutrophils (HI-N) | Clinical response for neutrophils was assessed as the number of participants achieving a hematological improvement - neutrophils (HI-N). Response was defined as an absolute increase in neutrophils > 0.5 × 10^9/L that was also an increase of ≥ 100%. | Posted | Count of Participants | Participants | 8 weeks |
|
|
|
| Secondary | Red Blood Cell (RBC) Transfusion Independence (RBC TI) | Clinical response for red blood cells was assessed as the number of participants who were transfusion dependent that achieve red blood cell (RBC) transfusion independence (RBC TI) for for 8 weeks or longer. | Posted | Count of Participants | Participants | 12 months |
|
|
|
| 1 |
| 3 |
| 3 |
| 3 |
| 3 |
| 3 |
| EG001 | Enasidenib Mesylat 200mg | Participants in this dose-escalation cohort self-administered enasidenib mesylate orally at a dose of 200 mg once daily. | 0 | 14 | 7 | 14 | 14 | 14 |
| Febrile Neutropenia | Blood and lymphatic system disorders | Systematic Assessment |
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| Chest pain - cardiac | Cardiac disorders | Systematic Assessment |
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| Blurred vision | Eye disorders | Systematic Assessment |
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| Duodenal Hemorrhage | Gastrointestinal disorders | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
|
| Localized Edema | General disorders | Systematic Assessment |
|
| Fatigue | General disorders | Systematic Assessment |
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| Volume overload | General disorders | Systematic Assessment |
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| Ammonium level elevated | Hepatobiliary disorders | Systematic Assessment |
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| Sepsis | Infections and infestations | Systematic Assessment |
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| Lung Infection | Infections and infestations | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
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| Hip Fracture | Injury, poisoning and procedural complications | Systematic Assessment |
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| Adult Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Pneumonitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Skin Infection | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Syncope | Nervous system disorders | Systematic Assessment |
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| Intracranial hemorrhage | Nervous system disorders | Systematic Assessment |
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| Edema Cerebral | Nervous system disorders | Systematic Assessment |
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| Generalized Muscle Weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Vasculitis | Vascular disorders | Systematic Assessment |
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| Elevated Unconjugated Bilirubin | Blood and lymphatic system disorders | Systematic Assessment |
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| Chest pain - cardiac | Cardiac disorders | Systematic Assessment |
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| Arthritis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Generalized muscle weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Muscle cramp | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Rash- Unspecified | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Bloating | Gastrointestinal disorders | Systematic Assessment |
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| Colitis | Gastrointestinal disorders | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| Gastritis | Gastrointestinal disorders | Systematic Assessment |
|
| Mucositis oral | Gastrointestinal disorders | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Stomach pain | Gastrointestinal disorders | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| Abdominal Pain | Gastrointestinal disorders | Systematic Assessment |
|
| Chills | General disorders | Systematic Assessment |
|
| Duodenal Ulcer | General disorders | Systematic Assessment |
|
| Edema Limbs | General disorders | Systematic Assessment |
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| Fatigue | General disorders | Systematic Assessment |
|
| Generalized Edema | General disorders | Systematic Assessment |
|
| Hemorrhoids | General disorders | Systematic Assessment |
|
| Localized Edema | General disorders | Systematic Assessment |
|
| Pain | General disorders | Systematic Assessment |
|
| Autoimmune disorder | Immune system disorders | Systematic Assessment |
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| Bilateral lower extremity cellulitis | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| COVID-19 | Infections and infestations | Systematic Assessment |
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| Lung infection | Infections and infestations | Systematic Assessment |
|
| Papulopustular rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Sinusitis | Infections and infestations | Systematic Assessment |
|
| Upper respiratory infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Urinary tract infection | Renal and urinary disorders | Systematic Assessment |
|
| Bruising | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | Systematic Assessment |
|
| Neutrophil count decreased | Investigations | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | Systematic Assessment |
|
| Bilirubin increased | Investigations | Systematic Assessment |
|
| Creatinine increased | Investigations | Systematic Assessment |
|
| Electrocardiogram QT corrected interval prolonged | Investigations | Systematic Assessment |
|
| Platelet count decreased | Investigations | Systematic Assessment |
|
| Weight loss | Investigations | Systematic Assessment |
|
| Anorexia | Metabolism and nutrition disorders | Systematic Assessment |
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| Dehydration | Metabolism and nutrition disorders | Systematic Assessment |
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| Hypernatremia | Metabolism and nutrition disorders | Systematic Assessment |
|
| Hypoalbuminemia | Metabolism and nutrition disorders | Systematic Assessment |
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| Hypocalcemia | Metabolism and nutrition disorders | Systematic Assessment |
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| Hypomagnesemia | Metabolism and nutrition disorders | Systematic Assessment |
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| Hyponatremia | Metabolism and nutrition disorders | Systematic Assessment |
|
| Hypophosphatemia | Metabolism and nutrition disorders | Systematic Assessment |
|
| Iron overload | Metabolism and nutrition disorders | Systematic Assessment |
|
| Dizziness | Nervous system disorders | Systematic Assessment |
|
| Dysgeusia | Nervous system disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Confusion | Psychiatric disorders | Systematic Assessment |
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| Insomnia | Psychiatric disorders | Systematic Assessment |
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| Acute kidney injury | Renal and urinary disorders | Systematic Assessment |
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| Chronic kidney injury | Renal and urinary disorders | Systematic Assessment |
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| Dysuria | Renal and urinary disorders | Systematic Assessment |
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| Hematuria | Renal and urinary disorders | Systematic Assessment |
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| Proteinuria | Renal and urinary disorders | Systematic Assessment |
|
| Urinary tract infection | Renal and urinary disorders | Systematic Assessment |
|
| Urine discoloration | Renal and urinary disorders | Systematic Assessment |
|
| Allergic Rhinitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Rash acneiform | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Skin hyperpigmentation | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Skin Infection | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Tinea Pedis - Skin Infection | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Facial Rash - Prurtic | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Hypertension | Vascular disorders | Systematic Assessment |
|
| Hypotension | Vascular disorders | Systematic Assessment |
|
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| D015470 | Leukemia, Myeloid, Acute |