All patients will receive open label thymidine and deoxycytidine

Mitochondrial DNA Depletion Syndrome 2 Myopathic Type

Thymidine Kinase 2 Deficiency

Mitochondrial DNA nucleotide precursors. Dose escalation: 130mg/kg/day x 14 days, 260 mg/kg/day x 14 days, and 400mg/kg/day as tolerated. Compounds are taken orally and divided into 3 doses daily.

Thymidine

Columbia University

Treatment of TK2 Deficiency With Thymidine and Deoxycytidine

Deoxythymidine and Deoxycytidine Treatment for Thymidine Kinase 2 (TK2) Deficiency

Number of participants with treatment-related elevated alanine aminotransferase (ALT) serum level relative to upper limit of normal (expressed as ratios) grade 3 or higher as defined by CTCAE 4.03.

Number of participants with treatment-related elevated aspartate aminotransferase (AST) serum level relative to upper limit of normal (expressed as ratios) grade 3 or higher as defined by CTCAE 4.03.

Number of participants with treatment-related elevated gamma-glutamyltransferase (GGT) serum level relative to upper limit of normal (expressed as ratios) grade 3 or higher as defined by CTCAE 4.03.

Blood lymphocyte count increased relative to upper limit or normal or decreased relative to lower limit of normal (expressed as ratios) grade 3 or higher as defined by CTCAE 4.03.

Serum creatinine level increased relative to upper limit of normal (expressed as ratios) grade 3 or higher as defined by CTCAE 4.03.

Number of patients with treatment related electrocardiogram (ECG) QT corrected interval (QTc) grade 3 or higher as defined by CTCAE version 4.03.

Patient-Reported Outcome Measurement Information System (PROMIS) Scale v1.0 - Gastrointestinal Diarrhea 6a score (score range 0-30 with higher scores indicating more severe diarrhea)

Time to mechanical ventilation, death, or both will be assessed.

Distance walked in meters over 6 minutes will be measured in ambulatory patient.

Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) score (0-64 point range with higher scores indicating better function) will be assessed in infants to assess motor function.

Hammersmith Functional Motor Scale Expanded (HFMSE) score (0-66 point range with higher scores indicating better function) will be measured in subjects >1 year-old.

Vital capacity (percent of predicted normal based on age and height) will be measure by spirometry

Number of hours per day that subjects use mechanical ventilation will be recorded.

Neuro Quality of Life (Neuro-QoL) short forms will be used to assess effects of muscle weakness on motor function and activities of daily living. In adults, Lower and Upper Extremity scales will be assessed (0-80 points with higher scores indicating better function).

Neuro Quality of Life (Neuro-QoL) forms will be used to assess effects of muscle weakness on motor function and activities of daily living. In pediatric subjects (<18 years-old), Lower and Upper Extremity scales will be assessed (0-160 points with higher scores indicating better function).

Suicidal ideation will be assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS), which contains 6 "yes" or "no" questions. Answer of "yes" to any question indicates possible suicide risk and answer of "yes: to questions 4, 5, or 6 indicates high-risk.

Since there are very few patients in the world with TK2 Deficiency, we may share de-identified data with other researchers who are treating patients with TK2 Deficiency

Michio Hirano, MD

Professor of Neurology

Muscular Dystrophy Association

Hospital Universitario 12 de Octubre

Instituto de Salud Carlos III

University of Seville

Medical Research Council Mitochondrial Biology Unit

Centro de Investigación Biomédica en Red Enfermedades Raras (CIBERER), Spain

Hospitales Universitarios Virgen del Rocío

Vall d'Hebron Research Institute

Open label treatment with thymidine and deoxycytidine

Breast Cancer

Bone Metastasis

Pain

chemoradiation

Rabin Medical Center, Beilinson Campus

Chemoradiation for Bone Metastasis

Chemoradiation With Capecitabine for Palliation of Pain From Bone Metastasis

Patients were asked to score their pain on a scale of 0 (no pain) to 10 (worst possible pain) before treatment and at 1, 2, 4, 8 and 12 weeks after treatment initiation.

Consumption of analgesics was evaluated by the physician using the 5-point WHO score, as follows: level 0, no analgesics required; level 1, non-narcotic analgesics required occasionally; level 2, non-narcotic analgesics required regularly; level 3, narcotic analgesics required occasionally; level 4, narcotic analgesics required regularly

Rabin Medical Center

Sheba Medical Center

Chemotherapy regimen (XP):
D1- D14 Capecitabine 1000 mg/m2 bid p.o. D1 Cisplatin 75 mg/m2 + NS 150mL MIV over 1hr repeat every 3 weeks

XT Regimen:
D1- D14 Capecitabine 1000 mg/m2 bid p.o. D1, D8 Genexol (Paclitaxel) 80 mg/m2 + D5W 500mL MIV over 3hrs Repeat every 3 weeks

First Line Chemotherapy

Capecitabine Plus Cisplatin Versus Capecitabine Plus Paclitaxel

Advanced or Recurrent Esophageal Squamous Cell Carcinoma

3.2 Overview of Study Design This study is a prospective, randomized, phase II study comparing response rate between patients with XP chemotherapy versus XG chemotherapy for patients with metastatic squamous cell carcinoma.
Chemotherapy regimen (XP):
D1- D14 Capecitabine 1000 mg/m2 bid p.o. D1 Cisplatin 75 mg/m2 + NS 150mL MIV over 1hr every 3 weeks
Chemotherapy regimen (XT):
D1- D14 Capecitabine 1000 mg/m2 bid p.o. D1, D8 Genexol (Paclitaxel) 80 mg/m2 + D5W 500mL MIV over 3hrs every 3 weeks

Capecitabine plus cisplatin(XP) versus capecitabine plus paclitaxel(XT)

Samsung Medical Center

Efficacy Comparison Study of Combination Regimens to Treat Advanced Esophageal Squamous Cell Carcinoma

A Randomized Phase II Trial of Capecitabine Plus Cisplatin (XP) Versus Capecitabine Plus Paclitaxel (XT) as a First-line Treatment for Advanced or Recurrent Esophageal Squamous Cell Carcinoma

thymidine/unwohlsein und ermüdung

Treatment of TK2 Deficiency With Thymidine and Deoxycytidine

Chemoradiation for Bone Metastasis

Efficacy Comparison Study of Combination Regimens to Treat Advanced Esophageal Squamous Cell Carcinoma

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