Dosimetric planning of radioiodine therapy on the basis of pharmacokinetic modeling

The program complex of pharmacokinetic modeling and dosimetric planning of radioiodine therapy on the basis of clinical diagnostic data is developed. For 16 patients with the diagnosis «diffuse toxic goiter» (Graves' disease) individual kinetic parameters of transport of the thyroid radiopharmaceutical taken orally are identified and calculations of the absorbed doses in the thyroid, the stomach, the blood tissue, and the periodic-depletion bladder are performed. Three approaches to purpose of activity of radiopharmaceutical and feature of individual dosimetric planning of radioiodine therapy are considered and analysed

Rôle de la chimiokine SDF-1/CXCL12 et de des récepteurs CXCR4 et CXCR7 dans la régulation de la quiescence des progéniteurs hématopoïétiques humains

LE KREMLIN-B.- PARIS 11-BU Méd (940432101) / SudocSudocFranceF

Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study

<div><p>Spinal muscular atrophy (SMA) is a monogenic disorder caused by loss of function mutations in the <i>survival motor neuron 1</i> gene, which results in a broad range of disease severity, from neonatal to adult onset. There is currently a concerted effort to define the natural history of the disease and develop outcome measures that accurately capture its complexity. As several therapeutic strategies are currently under investigation and both the FDA and EMA have recently approved the first medical treatment for SMA, there is a critical need to identify the right association of responsive outcome measures and biomarkers for individual patient follow-up. As an approved treatment becomes available, untreated patients will soon become rare, further intensifying the need for a rapid, prospective and longitudinal study of the natural history of SMA Type 2 and 3. Here we present the baseline assessments of 81 patients aged 2 to 30 years of which 19 are non-sitter SMA Type 2, 34 are sitter SMA Type 2, 9 non-ambulant SMA Type 3 and 19 ambulant SMA Type 3. Collecting these data at nine sites in France, Germany and Belgium established the feasibility of gathering consistent data from numerous and demanding assessments in a multicenter SMA study. Most assessments discriminated between the four groups well. This included the Motor Function Measure (MFM), pulmonary function testing, strength, electroneuromyography, muscle imaging and workspace volume. Additionally, all of the assessments showed good correlation with the MFM score. As the untreated patient population decreases, having reliable and valid multi-site data will be imperative for recruitment in clinical trials. The pending two-year study results will evaluate the sensitivity of the studied outcomes and biomarkers to disease progression.</p><p><b>Trial Registration</b>: ClinicalTrials.gov (<a href="https://clinicaltrials.gov/ct2/show/NCT02391831" target="_blank">NCT02391831</a>).</p></div

Baseline clinical and physical characteristics and of enrolled patients.

<p>Baseline clinical and physical characteristics and of enrolled patients.</p

Pulmonary function in SMA type 2 and 3 patients.

<p>Pulmonary function in SMA type 2 and 3 patients.</p

Electrophysiologic assessment of SMA type 2 and 3 patients.

<p>Electrophysiologic assessment of SMA type 2 and 3 patients.</p

Molecular SMA biomarkers.

<p>Molecular SMA biomarkers.</p

Psychomotor development of patients with SMA type 2 and 3.

<p>A: General motor development; B: Fine motor development; Values for ages displayed on histograms are median (min-max); * p ≤ 0.05.</p

CONSORT diagram.

<p>¹ As this manuscript was limited to baseline data, there were no withdrawal of participants. ² To be defined as “sitter” the patient must have a score ≥ 1 on item 9 of the MFM (“with support of one or both upper limbs maintain the seated position for 5 seconds”). For 3 patients the MFM could not be performed at baseline. 2 were classified as sitters since they had a score of 3 at the 6-month follow-up visit and 1 was retrospectively classified as a non-sitter according to patient files. ³ To be defined as “Ambulant” the patient must be able to walk 10 meters without human assistance or use of an ambulation device such as a cane or a walker.</p