araag2/TREC_Precision-Medicine huggingface dataset:
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Sorafenib for Prophylaxis of Leukemia Relapse in Allo-HSCT Recipients With FLT3-ITD Positive AML

Summary: The purpose of this study is to evaluate the efficacy of sorafenib for prophylaxis of leukemia relapse in allogeneic stem cell transplant (Allo-HSCT) recipients with FLT3-ITD positive acute myeloid leukemia (AML).

Detailed description: Internal tandem duplication of FMS-like tyrosine kinase 3 (FLT3-ITD) mutations have been reported in 20%-30% of patients with acute myeloid leukemia (AML). FLT3-ITD-positive AML patients have an inferior survival, primarily due to lower complete remission (CR) rate and higher relapse rate. Although allogeneic hematopoietic stem cell transplantation (allo-HSCT) improves the outcomes of some FLT3-ITD-positive AML, a significant number will suffer disease recurrence after allo-HSCT. Sorafenib, an inhibitor of multiple kinases including FLT3, has shown promising activity in FLT3-ITD-positive AML. Recent studies have shown that sorafenib monotherapy or in combination with chemotherapy are effective in attaining CR, but they do not have significant improvement in relapse. Currently, prophylactic use of sorafenib after allo-HSCT has been rarely reported, and whether it can improve outcomes of FLT3-ITD-positive AML remains unclear.

Eligibility: Inclusion Criteria:
  -  FLT3-ITD Positive AML
  -  Allo-HSCT Recipients
  Exclusion Criteria:
  -  cardiac dysfunction (particularly congestive heart failure)
  -  hepatic abnormalities (bilirubin ≥ 3 mg/dL, aminotransferase> 2 times the upper limit of normal)
  -  renal dysfunction (creatinine clearance rate < 30 mL/min)
  -  Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
  -  Patients with any conditions not suitable for the trial (investigators' decision)

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Samples From Leukemia Patients and Their Donors to Identify Specific Antigens

Summary: The purpose of this project is to develop a process to identify highly personalized antigens that are uniquely expressed by the patient's own leukemia cells that can be used for cellular immune therapy.

Detailed description: It is well known that tumor cells and leukemia cells express different surface structures (called antigens) that can serve as targets for cancer cell destruction by the immune system. Effective immune therapies are characterized by high specificity and low toxicity. One of the major obstacles impeding the use of these therapies as standard of care is the identification of good target antigens. In acute myeloid leukemia (AML) there is major patient to patient variation in leukemia antigens, so there is no universal AML cell target. Rather, each patient has a unique array of potential cell targets. Thanks to the rapid progress of new DNA/RNA sequencing technologies, the identification of these unique, patient-specific leukemia cell antigen-targets is now possible.
  The purpose of this project is to develop a process to identify highly personalized antigens that are uniquely expressed by the patient's own leukemia cells that can be used for cellular immune therapy.

Eligibility: Inclusion Criteria:
  -  Diagnosis of Leukemia with plan to receive a bone marrow transplant
  Exclusion Criteria:

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Prospective Study on the Feasibility of Plasma FLT3-Ligand Assay to Achieve a First Estimate of Its Prognostic Value on the Outcome of Patients Treated Intensively for Acute Leukemia

Summary: Despite advances in their classification and treatment, acute leukemia remain incurable disease for the majority of patients. It is necessary to identify new prognostic markers of survival and new therapeutic targets to improve prognosis. Some studies have shown that Fms-like tyrosine kinase 3-ligand (FLT3-L) could be interesting from this. A more recent study from our group testing a novel therapy in acute lymphoblastic leukemia, showed an increase of this marker in the blood of patients responding to treatment. The aim of our prospective, non-interventional study is to measure the plasma levels of FLT3-L at different times of the management of patients with acute lymphoblastic leukemia but also myeloid. For this, analyzes of the samples collected in the usual care will be conducted to study the relationship between the plasma concentration of FLT3-L and outcomes. If there is any link, the FLT3-L could serve as a predictor of treatment response.


Eligibility: Inclusion Criteria:
  -  Acute myelogenous leukemia diagnosis or lymphoblastic
  -  Patient intensively treated
  -  Patients who agreed to participate in the study and having signed the consent biocollection
  Non-Inclusion Criteria:
  -  Patient aged under 18

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Haploidentical Hematopoietic Stem Cell Transplantation for Acute Leukemias

Summary: This is a prospective observational cohort study of haploidentical transplantation with post-transplant cyclophosphamide for acute leukemias using reduced intensity conditioning for acute myeloid leukemia (AML) and myeloablative conditioning for acute lymphoblastic leukemia (ALL).

Detailed description: Hematopoietic stem cell transplantation (HSCT) is the most effective treatment for acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), with the lowest rates of relapse.
  Fertility rate in Brazil is falling, and only 25% of people born today will have a matched sibling donor. On the other hand, currently donor non-related to about 50% of patients enrolled in Brazilian Receptor Registry (REREME). Consequently, at least 35% of patients won't have a matched donor.
  The haploidentical transplantation is defined as a partially matched hematopoietic cell transplantation, using a partially matched family donor (parent, sibling or child). Haploidentical transplantation activity is growing worldwide, with results comparable matched unrelated donors.
  The objective of this study is to test the feasibility of haploidentical transplantation with post transplant cyclophosphamide for acute leukemias in a Brazil.

Eligibility: Inclusion Criteria:
  -  Acute lymphoblastic leukemia or acute myeloid leukemia
  Exclusion Criteria:
  -  Severe comorbidities
  -  Second transplant

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Microtransplantation in Older Patients With Acute Myeloid Leukemia

Summary: The purpose of this study is to see whether HLA-mismatched donor cells infusion with chemotherapy (microtransplantation，MST) could increase complete remission (CR) and improve survival in older patients with acute myeloid leukemia (AML)，the investigators conducted a prospective, multicenter clinical trial of HLA-mismatched MST to estimate outcomes and toxicities.


Eligibility: Inclusion Criteria:
  -  de novo acute myeloid leukemia
  -  age 60-90
  -  have HLA mismatched donor
  Exclusion Criteria:
  -  have no suitable donor or donor refused
  -  patient refused to accept donor cells

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Optimal Treatment Strategy Based on for Pediatric AML

Summary: The purpose of this study is to optimize therapy according to the known risk factors and treatment response in pediatric acute myeloid leukemia (AML)

Detailed description: I. Risk group assessment Favorable prognosis group: Low risk features + Good response
  Intermediate prognosis group:
  1. Low risk features + Delayed response-1
  2. Standard risk features + Good response
  3. Standard risk features + Delayed response-1
  Poor prognosis group:
  1. Any high risk features irrespective of treatment response
  2. Any delayed response-2 irrespective of risk features
  3. Any refractory state irrespective of risk features
  4. Any early relapse
  II. Chemotherapy Induction-1: Cytarabine + idarubicin Induction-2: High dose (HD) cytarabine + mitoxantrone Consolidation-1: Cytarabine + idarubicin Consolidation-2: HD cytarabine + etoposide Consolidation-3: HD cytarabine + mitoxantrone Consolidation-4: HD cytarabine + etoposide
  III. Allogeneic hematopoietic stem cell transplantation (HSCT) Favorable prognosis group: chemotherapy only Intermediate prognosis group: chemotherapy or HSCT with reduced intensity conditioning Poor prognosis group: HSCT with myeloablative conditioning

Eligibility: Inclusion Criteria:
  -  Patients who were newly diagnosed with de novo AML
  -  Patients who had recurrent cytogenetic abnormalities of AML even though the bone marrow blast percent is lower than 20%
  Exclusion Criteria:
  -  Acute promyelocytic leukemia
  -  Down syndrome AML
  -  Therapy-related AML
  -  AML developed from myelodysplastic syndrome or other marrow failure syndrome
  -  Isolated myeloid sarcoma without bone marrow involvement
  -  Patients who cannot undergo chemotherapy as scheduled due to serious complications at diagnosis

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Shotgun Sequencing in Diagnosing Febrile Neutropenia in Patients With Acute Myeloid Leukemia

Summary: This research trial studies the shotgun sequencing of blood samples in diagnosing febrile neutropenia in patients with acute myeloid leukemia. Studying samples of blood from patients with acute myeloid leukemia in the laboratory may help identify pathogens and accurately diagnose infections such as febrile neutropenia.

Detailed description: PRIMARY OBJECTIVES:
  I. To test the hypothesis that shotgun metagenomics is not inferior to standard of care diagnostics in the detection of pathogens in patients with febrile neutropenia.
  SECONDARY OBJECTIVES:
  I. To establish a microbiological diagnosis with known or unknown pathogens in patients in whom standard care failed to yield a pathogenic diagnosis.
  OUTLINE:
  Patients undergo collection of blood samples before and during the episode of febrile neutropenia for up to 6 weeks.

Eligibility: Inclusion Criteria:
  -  Patients with febrile neutropenia
  -  A primary diagnosis of acute myeloid leukemia (AML)
  Exclusion Criteria:
  -  Prior history of febrile neutropenia

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: A Study of SKLB1028 in Subjects With Relapsed/Refractory Acute Myeloid Leukemia

Summary: Patients will receive oral SKLB1028 for 28 days to study the side effects, tolerability and best dose for treating relapsed or refractory acute myeloid leukemia With FLT3 Mutations.

Detailed description: It is open-label, dose escalation study designed to characterize the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of orally administered SKLB1028 as a single agent given daily for 28 days. Cohorts of 3 patients receive SKLB1028 until dose limiting toxicity is noted (DLT). At that point cohorts will expand to 6 patients until MTD is determined. Patients not experiencing DLT or significant disease progression could continue receiving SKLB1028 up to 1 year.

Eligibility: Inclusion Criteria:
  -  Written informed consent must be provided.
  -  Males and females age ≥ 18 years;
  -  Histopathologically documented primary or secondary AML, as defined by WHO criteria, confirmed by pathology review at treating institution, meeting at least one of the following:
  1. Refractory to at least 1 cycle of induction chemotherapy, or
  2. Relapsed after at least 1 cycle of induction chemotherapy, or
  3. Patient is not, according to the clinical judgment of the Principal Investigator, a candidate for induction chemotherapy due to age, comorbidity, or other factors;
  -  Eastern Cooperative Oncology Group (ECOG) performance status of 0-3;
  -  In the absence of rapidly progressing disease, the interval from prior treatment to time of SKLB1028 administration should be at least 2 weeks for cytotoxic agents, or at least 5 half-lives for noncytotoxic agents;
  -  Serum creatinine ≤1.5 × ULN;
  -  Total serum bilirubin ≤ 1.5 × ULN unless considered due to Gilbert's syndrome or leukemic organ involvement;
  -  Serum AST or ALT ≤ 3.0 × ULN unless considered due to leukemic organ involvement;
  -  Females of childbearing potential and sexually mature males must agree to use a medically accepted method of contraception throughout the study;
  Exclusion Criteria:
  -  Histologic diagnosis of acute promyelocytic leukemia;
  -  Clinically active central nervous system leukemia;
  -  Persistent clinically significant toxicity from prior chemotherapy that is Grade 2 or higher;
  -  Bone marrow transplant within 100 days prior to study;
  -  Active, uncontrolled infection;
  -  Major surgery within 4 weeks prior to study;
  -  Radiation therapy within 4 weeks prior to study;
  -  Left ventricular ejection fraction ≤1 × ULN,or﹤50%. Clinically significant ECG QTc prolongation (Male: >450ms, Female: >470ms).Significant cardiac disease.
  -  Human immunodeficiency virus positivity;
  -  Active hepatitis B or C or other active liver disease;
  -  Women who are pregnant, lactating;
  -  Medical condition, serious intercurrent illness, or other extenuating circumstance that, in the judgment of the Principal Investigator, could jeopardize patient safety or interfere with the objectives of the study.

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Molecular Characterization of Acute Erythroid Leukemia (M6-AML) Using Targeted Next-generation Sequencing

Summary: Acute erythroid leukemia (AEL) is a morphologically distinct, infrequent (o5%) acute myeloid leukemia (AML) designed as M6 in the French- American-British (FAB) classification. The World Health Organization classification recognizes two subclasses, M6a, a leukemia with myeloid blast cells, and M6b, a very rare, purely erythroid AML. It may be difficult to distinguish between a myelodysplastic syndrome and AEL because of the erythroblastic proliferation, which is increased when dysplasia is present. No recurrent cytogenetic abnormality is specific of AEL and the prognosis is poor with a median survival of 17 months. A study of 14 genes in a series of 92 cases has shown that mutations are frequent in AEL and somewhat differ from the other AMLs by the lower and higher proportion of FLT3-ITD and TP53 mutations, respectively. Only three cases of AEL are reported in the TCGA database. To further characterize AEL, determine whether it constitutes a distinct class of AML and document the reasons for its poor prognosis, the investigators will search for molecular alterations in 40 M6a-AMLs using array comparative genomic hybridization (aCGH) and next-generation sequencing (NGS) of 106 genes known or suspected to have a role in myeloid malignancies or in erythrocyte differentiation.


Eligibility: Inclusion Criteria:
  -  Age>18 year
  -  Diagnosis of AML6
  -  Written consent obtained
  Exclusion Criteria:
  -  No written consent
  -  No frozen samples

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Invasives Aspergillosis in Acute Myeloid Leukemia

Summary: Patients with acute myeloid leukemia (AML) are at risk to develop severe infections whose invasive aspergillosis (IA). These infections are leading to an important morbidity and mortality. Antifungal prophylaxis is recommended by posaconazole for AML patients during neutropenia induced by induction chemotherapy. Their application is not uniform.

Detailed description: Invasive aspergillosis are frequent infections in hematological malignancy in particular during neutropenia induced by chemotherapy. Their incidence ranged between 5 to 25% according to the literature. Mortality may reach 30%. Our study described IA incidence in AML patients treated by intensive chemotherapy depending on antifungal prophylaxis by posaconazole. From 2009 to 2011, any patients received antifungal prophylaxis. From 2012 to 2015, patients received posaconazole during induction and salvage chemotherapy. During the all study period, efficacy of posaconazole is evaluated according to construction/demolition periods in hospital. All patients are hospitalized in High Efficiency Particulate Air (HEPA) filtration system.

Eligibility: Inclusion Criteria:
  -  > 18 years
  -  acute myeloid leukemia
  -  intensive chemotherapy (induction, consolidation, salvage, bone marrow transplantation)
  Exclusion Criteria:
  -  < 18 years
  -  pregnancy
  -  no intensive chemotherapy (palliative treatment, azacytidine...)

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

Summary: The use of genomics and functional translational studies is to identify and diagnose patients with precursor features of myeloid neoplasms and inherited bone marrow failure syndromes.

Detailed description: The pre-myeloid cancer and bone marrow failure clinic is an individualized medicine clinic conceptualized for the early detection of myeloid malignancies such as, myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN), MDS/MPN overlap syndromes and acute myeloid leukemia (AML). Using next-generation sequencing technologies, clonal hematopoietic stem cell changes can be detected early on in myeloid malignancies. These changes are currently classified as follows:
  1. Idiopathic cytopenias of unclear significance (ICUS).
  2. Clonal cytopenias of unclear significance (CCUS).
  3. Clonal hematopoiesis of indeterminate prognosis (CHIP).
  4. Bone marrow failure syndromes with risk for clonal myeloid transformation.
  5. Familial/Germline syndromes with increased risk for myeloid malignancies.
  This clinic blends next-generation sequencing technologies with functional studies to help identify, prognosticate and define management plans; including early intervention strategies, for affected patients.

Eligibility: Inclusion Criteria:
  Patients with:
  -  Idiopathic cytopenias of unclear significance (ICUS)
  -  Clonal hematopoiesis of indeterminate significance (CHIP)
  -  Age related clonal hematopoiesis (ARCH)
  -  Clonal cytopenias of unclear significance (CCUS)
  -  Marrow failure syndromes with myeloid malignancy predisposition- telomere dysfunction, chromosomal breakage disorders, etc.
  -  Germ line inherited syndromes with risk for malignant transformation
  -  Low Risk Myelodysplastic Syndromes (MDS)
  Exclusion Criteria:
  -  Patients who are unable to understand and sign the Informed Consent Form

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Phase 2 Study of Quizartinib in Participants With Acute Myeloid Leukemia (AML) FLT3 Internal Tandem Duplication (FLT3/ITD) Mutation

Summary: This is a Phase 2, multi-center, open-label, single-arm study to evaluate the efficacy, safety and pharmacokinetics of quizartinib monotherapy in Japanese subjects with FLT3-ITD positive refractory or relapsed acute myeloid leukemia.


Eligibility: Inclusion Criteria:
  -  AML patients in first relapse or refractory after all prior therapy
  -  Presence of the FLT3-ITD activating mutation in bone marrow or peripheral blood
  -  Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0 to 2
  Exclusion Criteria:
  -  Diagnosis of acute promyelocytic leukemia
  -  AML secondary to prior chemotherapy for other neoplasms.
  -  Persistent, clinically significant > Grade 1 non-hematologic toxicity from prior AML therapy
  -  Prior treatment with a FLT3 targeted therapy
  -  Active infection not well controlled by antibacterial, antifungal and/or antiviral therapy

Relevance to patient case: not relevant
Patient case: 13-year-old male with acute myeloid leukemia; genetic feature: FLT3

Clinical trial: Treatment Patterns and Key Healthcare Resource Use in Acute Myeloid Leukemia (AML) With or Without FMS-like Tyrosine Kinase-3 (FLT3) Mutation Study Based on Retrospective Chart Review

Summary: The purpose of this study is to retrospectively evaluate the treatment patterns and AML-related key healthcare resource use among AML patients, stratified by FLT3 mutation status, intensive chemotherapy (IC) eligibility, and relapsed or refractory (R/R) status.

Detailed description: The current study is a retrospective non-interventional study using real-world data collected from existing medical records to evaluate descriptively the treatment patterns and key healthcare resource use among AML patients with or without FLT3 mutation. The current study relies on secondary use of existing data, and there is no intervention involved. Patients who received the first AML treatment after the initial diagnosis, or were classified as relapsed/refractory (R/R), between January 1, 2013 and December 31, 2015 will be randomly selected to be included in this study, and the data from their existing medical records will be extracted. Eligible patients will be grouped based on FLT3 mutation status, intensive chemotherapy (IC) eligibility, and R/R status.
  For newly diagnosed patients, the index date will be defined as the initiation date of the first AML treatment following initial diagnosis. For the R/R patients, the index date will be defined as the date of the patient being classified as R/R. The study period will be the period from the index date to last follow-up date or death, whichever comes earlier. The endpoint measurements of this study are treatment patterns and key AML-related healthcare resources used during the study period.

Eligibility: Inclusion Criteria:
  -  Confirmed diagnosis of AML but NOT acute promyelocytic leukemia (APL)
  -  Known FLT3 mutation status
  -  Under the care of the participating physician during the past 3 years OR from the initial diagnosis of AML
  -  The medical records related to AML for the patient are available to the physician and can be abstracted for this study
  -  The medical records contains complete information on treatments and AML-related hospitalization, including admission date, length of stay, and reason of hospitalization
  -  Initiation date of first treatment after AML diagnosis OR date of being classified as relapsed from or being refractory to initial treatment (R/R) is between January 1, 2013 and December 31, 2015
  Exclusion Criteria:

Relevance to patient case: not relevant