araag2/TREC_Precision-Medicine huggingface dataset:
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Analysis of Genetic Factors Related to Predisposition and Prognosis of Hematological Malignancies in Israel

Summary: There are naturally occuring variations in the genetic makeup of all of us. Some of these variations may contribute to a change in susceptibility toward different diseases or change the prognosis.
  We are studying these genetic variations in patients with leukemia. The genes we are studying are those which influence detoxification of drugs and toxins.

Detailed description: We have prepared DNA on patients with leukemia (AML, CML and CLL) and we are studying the influence of polymorphisms in drug metabolism and disposition genes on predisposition and prognosis of these types of leukemia. The genes we are studying include: MDR1, CYP3A4, NQO1, NAT1/2, GST, hOCT. We plan to study other genes that may be contributory.
  Study includes comparing the frequency of polymorphisms in these genes with the frequency in the general population and the study of DNA biding proteins which bind at the sites of these polymorphisms.

Eligibility: Inclusion Criteria:
  -  diagnosis of acute myeloid leukemia or chronic lymphocytic leukemia
  Exclusion Criteria:
  -  under 18 years of age

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: WT1 for the Detection of Minimal Residual Disease

Summary: The purpose of this study is to determine if WT1 is an adequate measurement of minimal residual disease in leukemic patients.

Detailed description: Patients with acute Leukemia may have a large number of leukemic cells at the time that leukemia is evident clinically. At the time that we determine that a patient is in complete remission (CR) the patient may still have leukemic cells present in smaller quantities. One of the most important factors in the successful treatment of patients with leukemia is the ability to determine if the eradication of leukemia has been achieved. The determination of Minimal Residual Disease may be important in the determination of the therapy that a given patient will receive as determined by the level of residual disease.
  WT1 gene function and expression. The WT1 gene is a candidate gene for Wilms tumor, which is thought to arise as a result from inactivation of both alleles of the WT1 gene located at chromosome 11p13. The WT1 gene has been considered a tumor suppressor gene because intragenic deletions or mutations are found in tumors, germline mutations have been found in-patients with leukemia, and mediates growth suppression of Wilms tumor cells expressing a WT1 splicing variant.

Eligibility: Inclusion Criteria:
  -  Age less than 21 years of age.
  -  Patients with the following diseases: Acute lymphoblastic Leukemia (ALL) at the time of diagnoses or relapse, Acute non-lymphoblastic leukemia (ANLL) at diagnosis or after relapse and Chronic Myelogenous leukemia in chronic or accelerated phase.
  -  Patients will be eligible for any of the available treatment protocols or protocols for stem cell transplantation, regardless of the source of stem cells.
  -  Patients or legal guardians will sign an Institutional Review Board (IRB) approved informed consent.
  -  Patients will have venous access or peripheral vein for sampling.

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Allogeneic Transplantation for Patients With Acute Leukemia or Chronic Myelogenous Leukemia (CML)

Summary: The purpose of the study is to evaluate the overall and disease free survival of recipients who have received G-CSF mobilized stem cells from HLA matched sibling donors.

Detailed description: Allogeneic Peripheral Blood Stem Cell Transplantation for Patients with Acute Myelogenous Leukemia and Acute Lymphoblastic Leukemia in First Remission or Chronic Myelogenous Leukemia in First Chronic Phase

Eligibility: Inclusion Criteria:1. Any patient with one of the following hematologic malignancies in whom an allogeneic stem cell transplant is warranted: Specific disease categories include:
  1. acute myelogenous leukemia, 1st or 2nd remission
  2. acute lymphoblastic leukemia, 1st or 2nd remission
  3. chronic myelogenous leukemia, 1st or 2nd CP, accelerated phase 2. Patient age > 1 month and < 55 yo 3. Patients must have a genotypically HLA identical sibling 4. Patient must have adequate function as follows:
  a. total bilirubin <2.5 and SGOT/SGPT <2x normal b. adequate renal function as defined by creatinine < 1.5 or a 24 hr creatinine clearance >50 cc/min as determined by the Cockroft-Gault formula (to be done if serum creatinine > 1.5) c. DLCO > 60% predicted d. radionuclide cardiac scan with ejection fraction >45% 5. Patient must be competent to give consent.
  Inclusion criteria (Donor):
  1. HLA identical family member
  2. Donor or guardian must be competent to give consent
  3. Donor must have adequate veins for leukapheresis or agree to placement of central venous catheter
  Exclusion Criteria:3.2 Exclusion Criteria (Patient):
  1. Evidence of active infection or active hepatitis
  2. Positive serologies for HIV-1,HIV-2 or hepatitis B surface ag+
  3. Previous allogeneic stem cell/bone marrow transplant
  4. Pregnant or lactating patients
  4 Exclusion criteria (Donor):
  1. Donors who for psychologic, physiologic or medical reasons are unable to tolerate PBSC harvest
  2. Donors who are HIV+ or hepatitis B antigen +
  3. History of allergic reaction to G-CSF
  4. Female donors must be post-menopausal or have a negative pregnancy test

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Child-Parent Psychotherapy for Preschooler Witnesses of Domestic Violence Program

Summary: This study will examine the efficacy of Child-Parent Psychotherapy (CPP) for the treatment of preschoolers exposed to marital violence.

Detailed description: This study examines the efficacy of Child-Parent Psychotherapy (CPP) for the treatment of preschoolers exposed to marital violence. Multi-ethnic preschool-mother dyads from diverse socioeconomic backgrounds were randomly assigned to CPP or to a case management plus community referral for individual treatment comparison group. It was hypothesized the children who received CPP treatment would show significantly greater improvement in general symptomatology and in traumatic stress symptoms than those in the comparison group.
  There is growing recognition that, contrary to the long-standing assumption that young children are impervious to environmental stresses, preschoolers exposed to violence show increased rates of disturbances in self-regulation and in emotional, social and cognitive functioning (Osofsky, 2004; Pynoos et al., 1999; van der Kolk, 2003). The present study examines the efficacy of a relationship-based treatment approach involving the child and the mother. Dyads were randomly assigned to either the Child-Parent Psychotherapy (CPP) treatment group or to a comparison group that consisted of monthly case management by an experienced Ph.D.-level clinician plus referrals for individual treatment in the community for mothers and child. We hypothesized that Child-Parent Psychotherapy would be more effective in alleviating children's traumatic stress symptoms and behavior problems because it focuses on improving the quality of the child-mother relationship and engages the mother as the child's ally in coping with the trauma. Treatment was offered for 50 weeks.

Eligibility: Inclusion Criteria:
  -  child 3-5 years old
  -  child exposed to marital violence as confirmed by mother's report on the Conflict Tactics Scale 2 (Straus et al., 1996)
  -  perpetrator was not living in the home.
  Exclusion Criteria:
  -  mother's documented abuse of the target child
  -  current maternal substance abuse
  -  homelessness
  -  maternal mental retardation
  -  maternal psychosis
  -  child mental retardation or autistic-spectrum disorder

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Safety and Efficacy of Imatinib Added to Chemotherapy in Treatment of Ph+ Acute Lymphoblastic Leukemia in Children

Summary: The purpose of this study is to determine whether Imatinib is safe and effective in association with intensive treatment of Ph+ALL in children.

Detailed description: Recent advances in treatment have increased the cure of childhood ALL to 75% or better. However, attempts to improve results for resistant subtypes of ALL, such as Ph+ ALL, have been largely unsuccessful. Imatinib, an inhibitor of protein-tyrosine kinases, is currently being tested in several phase I, II and III trials covering most Chronic Myeloid Leukemia patient populations and patients with overtly relapsed or refractory Ph+ALL. Pediatric patients with Ph+ALL will receive Imatinib, added to intensive, post-induction BFM-type chemotherapy. The endpoint will be the evaluation on the long-term clinical outcome, in particular on the Disease Free Survival (DFS).

Eligibility: Inclusion Criteria:
  -  Children and adolescents aged 1 to 17 years at diagnostic
  -  Documented Ph+ ALL
  -  Eligibility for the current local prospective therapeutic study of childhood ALL
  -  Informed consent given by the parents or by legal guardian
  Exclusion Criteria:
  -  Abnormal hepatic functions
  -  Abnormal renal functions
  -  Active systemic bacterial, fungal or viral infection

Relevance to patient case: definitely relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Expanded Access Program of AMN107 in Imatinib-resistant or Intolerant Adult Patients With Chronic Myeloid Leukemia

Summary: This study will further evaluate if AMN107 is safe in adults with chronic myeloid leukemia who are resistant or intolerant to imatinib and to provide patients access to this new drug until the drug becomes commercially available.


Eligibility: Inclusion criteria:
  -  Imatinib-resistant or -intolerant Philadelphia chromosome-positive chronic myeloid leukemia in blast crisis, accelerated phase, or chronic phase.
  -  Chronic myeloid leukemia patients who have been treated with an investigational tyrosine kinase inhibitor.
  -  Laboratory values within normal limits.
  Exclusion criteria:
  -  Impaired cardiac function.
  -  Acute or chronic liver or renal disease considered unrelated to tumor.
  -  Other concurrent severe and/or uncontrolled medical conditions (e.g., uncontrolled diabetes, active or uncontrolled infection) that could cause unacceptable safety risks or compromise compliance with the protocol.
  -  Patients who are currently receiving treatment with any of the medications that have the potential to prolong the QT interval or to inhibit the metabolism of AMN107 (CYP3A4/5 inhibitors).
  -  Patients with a history of another malignancy that is currently clinically significant or currently requires active intervention.
  Other protocol-defined inclusion/exclusion criteria may apply.

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Study of Dasatinib in Children and Adolescents With Relapsed or Refractory Leukemia

Summary: The purpose of this clinical research study was to establish a recommended phase 2 once daily (QD) dose of dasatinib and to assess the efficacy of the investigational drug for relapsed or refractory (resistant to previous treatment) leukemia in children and adolescents. The side effects that this oral investigational drug may have in children, and the levels of the drug in the blood, will be studied at different doses.


Eligibility: For more information regarding BMS clinical trial participation, please visit www.BMSStudyConnect.com
  Inclusion Criteria:
  -  Ph-positive (Ph+) Chronic Myelogenous Leukemia in chronic, accelerated or blast phase or Ph+ acute lymphoblastic leukemia (ALL) with imatinib-resistant disease or intolerance to imatinib.
  -  Ph-negative acute leukemia in second or subsequent relapse
  -  Age >1 and <21 years
  -  Lansky or Karnofsky scale >60
  -  Life expectancy >3 weeks
  -  Adequate hepatic and renal function
  -  Written informed consent
  Exclusion Criteria:
  -  Subjects for whom potentially-curative therapy was available, including electing immediate [ie, planned <45 days] stem-cell transplantation. Subjects in Stratum 1 were to have had an ongoing identical HLA donor search, and may have discontinued study if a donor became available.)
  -  Subjects with symptomatic central nervous system (CNS) disease (eg, convulsions due to their CNS disease).
  -  Subjects who had not recovered from acute toxicity of previous therapy.
  -  Clinically-significant disorder of platelet function (eg, von Willebrand's disease) or ongoing gastrointestinal bleeding.
  -  Serious uncontrolled medical disorder or active infection
  -  Uncontrolled or significant cardiovascular disease
  -  Use of any investigational agent or any other anticancer agent within 14 days prior to treatment start.
  -  Prior therapy with dasatinib
  -  Subjects taking medications that irreversibly inhibit platelet function or anticoagulants.
  -  Subjects taking certain medications that are accepted to have a risk of causing QTc prolongation.
  -  Women of Child Bearing Potential with a positive pregnancy test prior to study drug administration.
  -  Expected noncompliance, or unable to have regular follow-up due to psychologic, social, familial, or geographic reasons.
  -  Subjects who are compulsorily detained for legal reasons or treatment of either a psychiatric or physical (eg, infectious disease) illness must not be enrolled into this study.

Relevance to patient case: definitely relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Osteoporosis in Children With ALL

Summary: Hypothesis:
  Pediatric patients with acute lymphoblastic leukemia, treated with chronic glucocorticoids as a part of the leukemia treatment protocol, will have an increased incidence and severity of osteoporosis.

Detailed description: Osteoporosis is a disease characterized by low bone mass and microarchitectural deterioration of bone tissue. Osteoporosis is a devastating disorder with significant physical, psychosocial and financial consequences. Intensive chemotherapy and radiotherapy have led to significant improvements in long term, disease-free survival of children with malignancies. Unfortunately, there are many deleterious side effects associated with this therapy. Little is known about the longitudinal changes in bone mass accumulation and bone metabolism in these patients.
  The purpose of this study is to evaluate the timing and severity of changes in bone mineral metabolism for children treated for childhood leukemia. Data will be used to establish treatment protocols with the goal of preventing severe fractures and pain in the acute treatment stage and severe osteoporosis and related pathology in the chronic stage.

Eligibility: Inclusion Criteria:
  -  All pediatric patients with a new diagnosis of ALL between ages 4-18
  -  Patients who are expected to receive glucocorticoids as part of the leukemia treatment protocol
  -  Bone density measurements must be performed at the diagnosis visit or maximally within 48 hours after the first dose of glucocorticoids
  Exclusion Criteria:
  -  Patients under 4 years of age are excluded due to lack of age appropriate standards for bone mineral density
  -  Glucocorticoid therapy of at least one month or longer within the six months prior to DX
  -  History of connective tissue or musculoskeletal disorders
  -  Biphosphonate therapy within the past 6 months
  -  Acute renal failure
  -  Serum creatinine equal to or higher than 4.5mg/dl
  -  Positive serum pregnancy test in adolescent females

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Natural Killer Index From Hematopoietic Stem Cell Graft

Summary: Numerous studies about the potential role of NK alloreactive during a n hematopoietic stem cells graft are based on genotypical analyses of the KIR receptors and on genotypic incompatibilities between KIR and HLA for couple donor/recipient. There is still a lot of issues non resolved: Are KIR really expressed and how occur their expression during time when hematopoietic reconstitution? Is it depending on HLA of the recipient?If KIR are expressed, what are the mechanisms of alloreactivity of NK cells? Are NK able to lyse tumoral cells? Could alloreactive NK cells constitute a therapeutic tool able to induce tolerance and elimination of leukemia during hematopoietic stem cells grafts?


Eligibility: Inclusion Criteria:
  -  Age superior to 1 year
  -  Patient that will be treated by an HSC graft
  -  Initial pathology of recipient: acute lymphoblastic leukemia, acute myeloblastic leukemia, chronic myeloid leukemia
  Exclusion Criteria:
  -  Patient already included in a study with an exclusion period
  -  HIV + or HCV + serology during pre-graft analysis
  -  Patient already treated by an allograft of HSC

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: A Phase I Safety, PK and PD Study of KW-2478 in Patients With Multiple Myeloma, Chronic Lymphocytic Leukaemia or B-cell Non-Hodgkin's Lymphoma

Summary: The aim of this study is to determine the safety, tolerability and dose-limiting toxicities of KW-2478 and to determine the Maximum Tolerated Dose and recommended Phase II dose for patients with relapsed/refractory MM, CLL or B-cell NHL.

Detailed description: This is a Phase I, open-label, dose-escalation study of KW-2478 in patients with relapsed/refractory multiple myeloma, chronic lymphocytic leukaemia or B-cell Non-Hodgkin's lymphoma who have no established therapeutic alternatives. Up to 42 patients will be enrolled at up to six investigational sites over a period of approximately 12 months until an MTD is reached. An additional 12 patients may be enrolled at the MTD in an expanded cohort of one or more of the eligible conditions.

Eligibility: Inclusion Criteria:
  1. Patients with a confirmed diagnosis of Multiple Myeloma, Chronic Lymphocytic Leukaemia, or B-cell Non-Hodgkin's Lymphoma, who have had at least two prior standard treatment regimens and are without established therapeutic alternatives.
  2. Signed IEC-approved informed consent
  3. ECOG performance status of 0, 1 or 2;
  4. Life expectancy of at least 3 months;
  5. Adequate haematologic status, liver function and renal function
  6. Patients of reproductive potential must agree to follow accepted birth control methods during the study
  Exclusion Criteria:
  1. No anti-cancer treatment for ≥ 3 weeks prior to receiving study drug
  2. Any other severe, acute or chronic illness
  3. No other prior or concurrent malignancy
  4. Immunosuppressant therapy

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Chromosome Abnormalities in Chronic Myeloid Leukemia (CML) on Imatinib. GIST Patients on Imatinib

Summary: In order to distinguish between clonal instability driven by imatinib in CML and actual changes with secondary clones induced by imatinib we would like to investigate the karyotype of non-CML patients treated with imatinib such as GIST patients.

Detailed description: Chronic myeloid leukemia (CML) is a myeloproliferative disorder characterized by the presence of the Philadelphia (Ph) chromosome - a t(9:22) translocation that results in the production of a BCR/ABL fusion protein with Abl kinase activity.
  Imatinib mesylate (Gleevec) specifically targets a limited set of protein tyrosine kinases - ABL, Arg (Abl-related gene), c-Kit, platelet-derived growth factor receptor (PDGF-R) - and their oncogenic forms, most notably BCR/ABL Imatinib is also a potent inhibitor of a receptor-type c-Kit tyrosine kinase. Therefore imatinib was examined for therapeutic efficacy against malignant gastro-intestinal stromal tumors (GIST) Recent articles have drawn attention to the development of new Ph-negative, cytogenetically unrelated clones after therapy of Ph-positive CML with imatinib. Trisomy 8 and monosomy 7 are the most frequent defects, but other aberrations have also been reported. Some of these cytogenetic abnormalities are associated with acute myeloid leukemia and MDS.

Eligibility: Inclusion Criteria:
  -  GIST patient on Imatinib for more than 12 months
  Exclusion Criteria:

Relevance to patient case: not relevant
Patient case: 4-year-old female with leukemia; genetic feature: ABL1

Clinical trial: Vaccine Therapy in Treating Patients Who Have Undergone a Donor Stem Cell Transplant and Have Cytomegalovirus Infection That Has Not Responded to Therapy

Summary: RATIONALE: Vaccines may help the body build an effective immune response to kill cytomegalovirus infections.
  PURPOSE: This phase I trial is studying the side effects and best dose of vaccine therapy in treating patients who have undergone a donor stem cell transplant and have cytomegalovirus infection that has not responded to therapy.

Detailed description: OBJECTIVES:
  Primary
  -  To determine the safety of infusing cytomegalovirus (CMV) pp65-specific cytotoxic T-lymphocytes (CTL) generated using pp65 peptides in patients who have undergone allogeneic stem cell transplantation and have persistent CMV infections.
  Secondary
  -  Characterize CMV pp65-specific immune responses in terms of cytotoxicity and cytokine production pre-infusion and then periodically thereafter.
  -  Characterize the levels of CMV DNA in recipients of CMV pp65 CTL and observe whether the CTL infusion has any impact on the level of virus.
  OUTLINE: This is a multicenter study.
  Patients receive cytomegalovirus (CMV) pp65 cytotoxic T-cell infusion on day 1. Patients may receive up to 2 more doses at least 2 weeks after previous dose.
  Blood samples are collected and analyzed by quantitative CMV PCR, chromium release assays for CMV pp65-specific cytotoxicity, and immunophenotype for CD3, CD4, CD8, CD56, CD19, and CD45 RA/RD. Intracellular cytofluorometry is used to assess IL-2, IL-4, IL-10, and IFN-γ production by CD4 and CD8 CMV-specific effector cells.
  After completion of study treatment, patients are followed periodically for up to 1 year.

Eligibility: DISEASE CHARACTERISTICS:
  -  Cytomegalovirus (CMV) seropositive
  -  Patient has had CMV antigenemia for ≥ 2 weeks OR CMV DNA levels ≥ 600 copies/μg of DNA despite antiviral therapy targeting CMV (ganciclovir or foscarnet)
  -  No prior allogeneic stem cell transplantation before the most recent transplantation
  -  CMV seropositive donor negative for HIV-1, HIV-2, HTLV-1/2 available
  PATIENT CHARACTERISTICS:
  -  ECOG performance status 0-2 (for patients ≤ 16 years of age) OR Lansky performance status 70-100%
  -  Bilirubin < 2.0 mg/dL
  -  AST and ALT < 2.5 times upper limit of normal
  -  Creatinine clearance > 50 mL/min
  -  Pulse oximetry > 95% without supplemental oxygen
  -  No history of graft-vs-host disease (GVHD) ≥ grade 2
  -  Not moribund
  -  No patients not expected to survive 1 month after T cell infusion due to cardiac, pulmonary, renal, hepatic, or neurologic dysfunction
  PRIOR CONCURRENT THERAPY:
  -  No concurrent systemic immunosuppressive agents for the treatment of GVHD

Relevance to patient case: not relevant