High-Dose Riboflavin as a Prophylactic Treatment of Migraine: Results of an Open Pilot Study

Cephalalgia ◽  
1994 ◽  
Vol 14 (5) ◽  
pp. 328-329 ◽  
Author(s):  
J Schoenen ◽  
M Lenaerts ◽  
E Bastings
Keyword(s):  
Pilot Study ◽  
Side Effects ◽  
Prophylactic Treatment ◽  
Low Cost ◽  
Controlled Trial ◽  
High Dose ◽  
Short Term ◽  
Global Improvement ◽  
The Brain ◽  
Open Pilot

If the brain of migraineurs is characterized between attacks by a reduction of mitochondrial phosphorylation potential, riboflavin, which has the potential of increasing mitochondrial energy efficiency, might have prophylactic effects in migraine. In this preliminary open pilot study, 49 patients suffering from migraine (45 without aura, 4 with aura) were treated with 400 mg of riboflavin as a single oral dose for at least 3 months. Twenty-three patients received in addition 75 mg of aspirin. Mean global improvement after therapy was 68.2% and there was no difference between the two groups of patients. With the exception of one patient in the riboflavin plus aspirin group who withdrew because of gastric intolerance, no drug-related side effects were reported. High-dose riboflavin could thus be an effective, low-cost prophylactic treatment of migraine devoid of short-term side effects. A placebo-controlled trial of its efficacy seems worthwhile.

Metoclopramide Treatment in Patients with Diamond-Blackfan Anemia: Preliminary Results of a French Prospective Trial.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 2843-2843
Author(s):  
Thierry Leblanc ◽  
Isabelle Marie ◽  
Marianne Debre ◽  
Lydie Da Costa ◽  
Pierre Bordigoni ◽  
...  
Keyword(s):  
Pilot Study ◽  
Side Effects ◽  
Low Cost ◽  
Accurate Determination ◽  
Prospective Trial ◽  
Drug Efficacy ◽  
Complete Response ◽  
High Dose ◽  
Diamond Blackfan Anemia ◽  
Nervous Breakdown

Abstract Metoclopramide has been shown to be a potential new therapeutic approach for Diamond-Blackfan anemia (DBA) affected individuals and has been hypothetized to stimulate erythropoiesisis through the induction of prolactin release. Among 15 transfusion-dependent patients enrolled in a previous pilot study, 9 completed 16 weeks of treatment, 3 of whom reaching a complete and sustained response (Abkowitz et al, Blood 2002). In order to further assess the drug efficacy we performed a prospective study in patients included in the French DBA registry. Patients over 2 years of age and dependent on regular transfusions were randomized at inclusion either to be treated immediately after a transfusion (Arm B), or 6 weeks later (Arm A), the comparison of these 2 groups aiming to provide a more accurate determination in the delay of response. All patients were prescribed metoclopramide for 16 or 22 weeks: 10 mg x 3/d in adults and 0.2 mg/kg x 3/d in children (weight < 50 kg). Packed Red Blood Cell transfusions were prescribed for hemoglobin values < 80g/L. The definition of response relied on Hb levels, reticulocyte counts, and the intervals between transfusions. Prolactin as well as other hormones levels were assessed. The study was initiated in October 2003. 7 adults and 22 children have been included so far (13F, 16M): 17 in A and 16 in B arms, respectively; 4 additional pts are planned to be included within a month. The treatment had to be prematurely discontinued in 2 pts because of severe side effects: nervous breakdown (8w of treatment) and anaphylaxis (14w of treatment). Other side effects were mild. Most patients complained of asthenia and drowsiness which both decreased after the 1st weeks of treatment; amenorrhoea (2 pts) and bulimia (3 pts) were also observed. No increase in transfusion need occurred. An update of the trial was performed on August 1st: at that time 21 pts had completed at least 16w of treatment. None of them experienced a complete response, as defined by a transfusion independence. In two a partial response, as defined by an increase in the transfusion interval was obtained: In one patient it evolved from 3–4 to 8 w, and in the other from 6–8 to 10–11w. Both are still on treatment. Lastly, it has to be emphasized that the 2 French responder patients previously included in the pilot study are still transfusion independent more than 5 yr after the initiation of metoclopramide treatment. CONCLUSION: metoclopramide may be of interest in some DBA affected individuals, even though we only obtained a low rate of partial responses in the present ongoing trial. We believe that this non toxic, and low cost treatment warrants to be tested in all patients with regular transfusion need or requiring high dose steroids. The duration of response and long term tolerance remain to be determined. Acknowledgments: Maria Daniela Arturi Foundation, DBA foundation and Assistance Publique-Hôpitaux de Paris (DRRC)

Effectiveness of Lamotrigine in the Prophylaxis of Migraine with Aura: an Open Pilot Study

Cephalalgia ◽  
1999 ◽  
Vol 19 (1) ◽  
pp. 64-66 ◽  
Author(s):  
G D'Andrea ◽  
F Granella ◽  
M Cadaldini ◽  
GC Manzoni
Keyword(s):  
Pilot Study ◽  
Migraine With Aura ◽  
High Frequency ◽  
Controlled Trial ◽  
Open Pilot

We report a small open pilot study to evaluate the efficacy of lamotrigine (100 mg/day) in the prevention of migraine with aura attacks. We studied 24 patients affected by migraine with aura with a high frequency of attacks. Following a 1-month run-in period, the patients took lamotrigine for 3 months. Mean attack number per month was reduced from 6.1 ± 4.1 during the run-in period to 0.7 ± 1.3 at the 3rd month of treatment ( p< 0.0001). In 13 out of 21 patients who completed the study, the attacks were completely abolished at the 3rd month of treatment, while only one patient was completely unresponsive to the drug. Lamotrigine seems worthy of a controlled trial as prophylaxis of a migraine with aura.

DHA supplementation in infants born preterm and the effect on attention at 18 months’ corrected age: follow-up of a subset of the N3RO randomised controlled trial

2020 ◽  
pp. 1-12 ◽  
Author(s):  
Erandi Hewawasam ◽  
Carmel T. Collins ◽  
Beverly S. Muhlhausler ◽  
Lisa N. Yelland ◽  
Lisa G. Smithers ◽  
...  
Keyword(s):  
Randomised Controlled Trial ◽  
Primary Outcome ◽  
Controlled Trial ◽  
High Dose ◽  
Peak Period ◽  
Soya Oil ◽  
Randomised Controlled ◽  
To Receive ◽  
The Brain

Abstract Infants born preterm miss out on the peak period of in utero DHA accretion to the brain during the last trimester of pregnancy which is hypothesised to contribute to the increased prevalence of neurodevelopmental deficits in this population. This study aimed to determine whether DHA supplementation in infants born preterm improves attention at 18 months’ corrected age. This is a follow-up of a subset of infants who participated in the N3RO randomised controlled trial. Infants were randomised to receive an enteral emulsion of high-dose DHA (60 mg/kg per d) or no DHA (soya oil – control) from within the first days of birth until 36 weeks’ post-menstrual age. The assessment of attention involved three tasks requiring the child to maintain attention on toy/s in either the presence or absence of competition or a distractor. The primary outcome was the child’s latency of distractibility when attention was focused on a toy. The primary outcome was available for seventy-three of the 120 infants that were eligible to participate. There was no evidence of a difference between groups in the latency of distractibility (adjusted mean difference: 0·08 s, 95 % CI –0·81, 0·97; P = 0·86). Enteral DHA supplementation did not result in improved attention in infants born preterm at 18 months’ corrected age.

scholarly journals Impact of the “BALatrine” Intervention on Soil-Transmitted Helminth Infections in Central Java, Indonesia: A Pilot Study

2019 ◽  
Vol 4 (4) ◽  
pp. 141
Author(s):  
Darren J Gray ◽  
Johanna M Kurscheid ◽  
MJ Park ◽  
Budi Laksono ◽  
Dongxu Wang ◽  
...  
Keyword(s):  
Pilot Study ◽  
Cumulative Incidence ◽  
Community Education ◽  
Low Cost ◽  
Controlled Trial ◽  
Cluster Randomised Controlled Trial ◽  
Intervention Village ◽  
Central Java ◽  
Control Village ◽  
Randomised Controlled

Many latrine campaigns in developing countries fail to be sustained because the introduced latrine is not appropriate to local socio-economic, cultural and environmental conditions, and there is an inadequate community health education component. We tested a low-cost, locally designed and constructed all-weather latrine (the “BALatrine”), together with community education promoting appropriate hygiene-related behaviour, to determine whether this integrated intervention effectively controlled soil-transmitted helminth (STH) infections. We undertook a pilot intervention study in two villages in Central Java, Indonesia. The villages were randomly allocated to either control or intervention with the intervention village receiving the BALatrine program and the control village receiving no program. STH-infection status was measured using the faecal flotation diagnostic method, before and eight months after the intervention. Over 8 months, the cumulative incidence of STH infection was significantly lower in the intervention village than in the control village: 13.4% vs. 27.5% (67/244 vs. 38/283, p < 0.001). The intervention was particularly effective among children: cumulative incidence 3.8% (2/53) for the intervention vs. 24.1% (13/54) for the control village (p < 0.001). The integrated BALatrine intervention was associated with a reduced incidence of STH infection. Following on from this pilot study, a large cluster-randomised controlled trial was commenced (ACTRN12613000523707).

Use of Cannabis for Agitation in Patients With Dementia

2020 ◽  
Vol 35 (7) ◽  
pp. 312-317
Author(s):  
Amanda Mueller ◽  
Danielle R. Fixen
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Side Effects ◽  
Older People ◽  
Endocannabinoid System ◽  
First Line ◽  
Short Term ◽  
Therapeutic Benefits ◽  
The Brain

Studies have reported changes in the endocannabinoid system in the brain of patients with Alzheimer's disease (AD), playing a role in the pathophysiology of AD. Cannabinoids have been shown to have neuroprotective properties, reduce neuroinflammation, and enhance neurogenesis. Evidence suggests that the utilization of marijuana products containing both tetrahydrocannabinol (THC) and cannabidiol (CBD) or CBD alone have been effective and safe for use in older people with agitation associated with dementia. A review in 2017 summarized positive findings for therapeutic benefits of cannabinoids in agitation of AD and dementia, but there was no definitive conclusion because of varying cannabinoid products. Cannabinoids were shown to be well tolerated, with few short-term side effects. This differs from first-line medications utilized for dementia behaviors, which can have unwanted side effects. Further research regarding the safety, efficacy, and variability of these products in older people is needed.

scholarly journals The effect of short-term, high-dose oral N-acetylcysteine treatment on oxidative stress markers in cystic fibrosis patients with chronic P. aeruginosa infection — A pilot study

2015 ◽  
Vol 14 (2) ◽  
pp. 211-218 ◽  
Author(s):  
Marianne Skov ◽  
Tacjana Pressler ◽  
Jens Lykkesfeldt ◽  
Henrik Enghusen Poulsen ◽  
Peter Østrup Jensen ◽  
...  
Keyword(s):  
Oxidative Stress ◽  
Cystic Fibrosis ◽  
Pilot Study ◽  
High Dose ◽  
Oxidative Stress Markers ◽  
Short Term ◽  
Stress Markers ◽  
Dose Oral

scholarly journals Pilot Study of Threonine Supplementation in Human Spasticity

1982 ◽  
Vol 9 (2) ◽  
pp. 141-145 ◽  
Author(s):  
A. Barbeau ◽  
M. Roy ◽  
C. Chouza
Keyword(s):  
Pilot Study ◽  
Side Effects ◽  
Motor Performance ◽  
Pyramidal Tract ◽  
Controlled Trial ◽  
Lower Limbs ◽  
Upper Limbs ◽  
Muscle Spasms ◽  
Objectively Measured ◽  
Observation Period

ABSTRACT:Threonine supplementation (500 mg/day) was given to 6 patients with genetic spasticity syndromes for a period of 12 months, followed by a 4-month observation period without medication. All 6 patients showed partial improvement of spasticity, intensity of knee jerks and muscle spasms without changes in true pyramidal tract signs. The improvement in motor performance, objectively measured, averaged 29% (19% in upper limbs and 42% in lower limbs). The range of overall improvement was 19–35% (7–30% for upper limbs; 25–67% for lower limbs). No toxic clinical or biochemical side effects were encountered. Thus threonine, a precursor of glycine, produced the same effect on spasticity than that previously observed with glycine. It is concluded that threonine supplementation is feasible and safe and that it deserves a controlled trial in well defined (preferably genetic) cases of spasticity.

scholarly journals Assessing Patient Preferences for the Benefits and Risks of Treating for Acute Myeloid Leukemia (AML): A Pilot Study

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4799-4799 ◽  
Author(s):  
Jaein Seo ◽  
B. Douglas Smith ◽  
Elihu H. Estey ◽  
Ernest S. Voyard ◽  
Bernadette O'Donoghue ◽  
...  
Keyword(s):  
Pilot Study ◽  
Side Effects ◽  
Patient Preferences ◽  
Myeloid Leukemia ◽  
Research Funding ◽  
Z Score ◽  
Short Term ◽  
Stakeholder Committee ◽  
Society Research

Abstract Introduction:Acute Myeloid Leukemia (AML) is a blood cancer which progresses rapidly in the absence of treatment. There have been few advances in the treatment of AML over the last three decades. In 2015 the Leukemia and Lymphoma Society (LLS) initiated a research program to assess patient preferences for AML treatments. The aim of this research was to promote patient-focused drug development and inform future regulatory decisions. We sought to develop and pilot a patient-centered survey instrument to assess patient preferences for the benefits and risks of AML treatments. Methods: Development was informed by a targeted literature review and engagement with an expert stakeholder committee (n=12) to guide the clinical accuracy and relevance of the survey instrument. A community stakeholder committee, consisting of patients with AML and caregivers (n=15), provided information about their experiences with AML and various treatments. They also engaged in pretest interviews to test comprehension and ensure it captured the patient experience. A discrete-choice experiment (DCE) was developed spanning 5 benefits and risks, including event-free survival (EFS), complete remission (CR), time in hospital, short-term side effects, and long-term side effects. This DCE consisted of 16 pairs of hypothetical treatments, with participants being asked to identify which treatment they would prefer in each pair. Results of a pilot study with AML patients and caregivers were assessed by Z-score that were derived from a conditional logistic model regressing each attribute upon their choices. Results: The pilot included 18 patients and 8 caregivers with a mean age of 50 years (range=24-81). Most participants were college educated (n=22), Caucasian (n=19), privately insured (n=21), and employed (n=13). Participants valued CR the most (Z-score=7.95, p<0.001), followed by EFS (5.32, p<0.001). They were most averse to time in hospital (-3.41, p=0.001), followed by long-term side effects (-3.03, p=0.002) and short-term side effects (-1.99, p=0.047), which was marginally significant. Conclusions: This study demonstrates the value of rigorous community engagement in developing survey instruments to measure patient preferences. The results of this pilot study demonstrate the ability of our DCE to measure treatment preferences of AML patients and caregivers. Given this success, we are currently engaged in a nationally study where we will recruit a larger and more diverse sample. The results of this national study will inform drug developers and regulatory decision makers. Disclosures Seo: The Leukemia & Lymphoma Society: Research Funding. Voyard:The Leukemia & Lymphoma Society: Employment. O'Donoghue:The Leukemia & Lymphoma Society: Employment. Bridges:The Leukemia & Lymphoma Society: Research Funding.

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