Pilot Safety Research Articles

Feasibility of Minocycline and Doxycycline Use as Potential Vasculostatic Therapy for Brain Vascular Malformations: Pilot Study of Adverse Events and Tolerance

Background: Tetracyclines may be useful in preventing pathological vascular remodeling, thus decreasing the risk of spontaneous hemorrhage from brain vascular malformations. Methods: Arteriovenous malformation (AVM) and intracranial aneurysm patients undergoing noninvasive management were treated with minocycline or doxycycline (200 mg/day) up to 2 years in a prospective open-label safety pilot trial. The primary outcome was to compare dose-limiting intolerance, defined as treatment-related dose reduction or withdrawal between the agents.Results: Twenty-six patients with AVMs (n = 12) or aneurysms (n = 14) were recruited. Adverse event rates were similar to other reported trials of these agents; 4 of 13 (31%) minocycline and 3 of 13 (23%) doxycycline patients had dose-limiting intolerance (hazard ratio = 3.1, 95% CI = 0.52–18.11, log rank p = 0.70). Conclusions: It is feasible to propose a long-term trial to assess the potential benefit of tetracycline therapy to decrease hemorrhagic risk in brain vascular malformations.

Possibilities of Improving Pilot's Situational Awareness through the Shaping of the Human-Machine Interface in Military Aircraft Cockpits

Possibilities of Improving Pilot's Situational Awareness through the Shaping of the Human-Machine Interface in Military Aircraft CockpitsWith determined individual perception capability, the pilot's safety during a flight depends on his situational awareness formed by information from the surrounding environment. The graphical data information may be modeled and performed to the pilot by a data interface of research simulator. The method of testing human-machine interface (HMI)described in this paper enables detecting special incidents (critical situations) influencing flight safety, and help making prevention efforts. The proposed research method can result in developing solutions for shaping the data interface, as well as ergonomic solutions in cockpits of military aircraft.

Master training in Agribusiness and Rural Development at the University of Zagreb

The title of the proposed JEP project is: “Agribusiness Higher EducAtion Development” with the acronym AHEAD. This curriculum development project – in case of acceptance – will last for three years, from July 2005 and June 2008. The primary project site is the University of Zagreb, Croatia; the contractor and the co-ordinator institution is the University of Debrecen, Hungary. In the consortium, 3 further European universities (University of Hohenheim, Wageningen University and Scottish Agricultural College) will participate, from the Croatian side the Ministry of Agriculture, Forestry and Water Management and an additional 7 Croatian institutes will also be involved. The total number of members in the consortium is 13. The main objective of the AHEAD project is to establish new BSc and MSc programmes in Croatia at two Faculties of the University of Zagreb. These are as follows: Agricultural and Rural Development, Food Safety and Quality Management and pilot MSc training in Agribusiness and Rural Development. These are preceded by faculty retraining programmes in food safety and quality management, as well as agribusiness and commerce within the framework of a MBA programme accredited by the International MBA Network. The professional content of the project is a modernised curriculum and training palette that would be available by the end of this project at the University of Zagreb, serving not only the higher education of the country, but the demand of the Croatian national economy as well, in line with the basic principles of the European Union.

Simvastatin in the acute phase of ischemic stroke: a safety and efficacy pilot trial

Although statins are being used for secondary prevention of ischemic stroke, recent experimental data have shown new pleiotropic effects of these drugs responsible for their role in neuroprotection. We conducted a pilot, double-blind, randomized, multicenter clinical trial to study for the first time safety and efficacy of simvastatin in the acute phase of ischemic stroke. Simvastatin/placebo was given at 3-12 h from symptom onset to 60 patients with cortical strokes. Efficacy on the evolution of several inflammation markers [interleukin (IL)-6, IL-8, IL-10, monocyte chemoattractant protein-1, intercellular adhesion molecule-1, vascular cell adhesion molecule-1, C-reactive protein, sApo/Fas, tumor necrosis factor-alpha, E-selectin, L-selectin and nitrites+nitrates] and neurological outcome was evaluated at baseline, day 1, 3, 5, 7 and 90. No differences were found amongst the biomarkers studied regarding treatment allocation. Although simvastatin patients improved significantly by the third day (46.4% vs. 17.9%, P = 0.022), a non-significant increase in mortality and greater proportion of infections (odds ratio 2.4, confidence interval 1.06-5.4) in the simvastatin group were the main safety concerns. Therefore, a larger clinical trial is needed to confirm the net benefit of this therapeutic approach.

Is there a role for hysteroscopic tubal occlusion of functionless hydrosalpinges prior to IVF/ICSI in modern practice?

To determine whether hysteroscopic tubal occlusion will produce the same efficacy as laparoscopic tubal occlusion of functionless hydrosalpinx prior to IVF/ICSI. A prospective comparative study. Setting. Endoscopy Unit of the Women's Health Center, Faculty of Medicine, Assiut University, Assiut, Egypt. A pilot safety phase included 10 uteri removed by hysterectomy in perimenopausal women subjected to roller ball coagulation of the peritubal bulge. The study phase included 27 patients with uni- or bilateral functionless hydrosalpinges, who were randomly divided into 2 groups. Group A comprised 14 patients who were randomly allocated for laparoscopic occlusion. Group B included 13 patients scheduled for a hysteroscopic approach. Interventions. Laparoscopic occlusion of the isthmic part of the fallopian tube was carried out using bipolar diathermy in 9 (64%) cases or clips in 3 (21.4%) cases in Group A. Roller ball electrode of the resectoscope was utilised for occlusion of the tubal ostium under local, spinal, or general anesthesia in Group B. Second-look office hysteroscopy was performed in Group B whenever possible. In both groups, hysterosalpingography or sonohysterography was carried out 1 month later to confirm tubal occlusion. Safety phase aimed at confirming tubal occlusion with minimal harm to adjacent tissues. Confirmed tubal occlusion of the functionless hydrosalpinx. The safety phase resulted in bilateral complete occlusion of the proximal part of the tubes with secondary coagulation <8 mm, as shown in the histopathologic sections. The suspected main cause of functionless hydrosalpinges was iatrogenic (pelvic surgery) in 9 (64%) and 8 (61.5%) cases in both groups, respectively. The mean number of abdominal scars/patient was 1.4 and 1.5 in both groups, respectively. Unilateral functionless hydrosalpinx was encountered in 7 (50%) and 5 (38%) cases in both groups, respectively. In Group A, the procedure was possible and successful in 10 cases (76.9%), while in Group B, hysteroscopic access and occlusion were achieved in 12 (85.7%) and 9 (64.2%) cases, respectively. In Group B, diagnostic hysteroscopy showed fine marginal adhesions in 2 cases (15%), and a small polyp in 1 case (7.7%). Hysteroscopic tubal occlusion showed shorter operative time (9+/-2.8 versus 24+/-4.8 min, p=0.0001) and hospital stay (2+/-1.8 versus 5+/-1.1h, p=0.0001). Second-look office hysteroscopy was performed in 8 cases in Group B and revealed no significant corneal lesions at the site of hysteroscopic occlusion. This preliminary study demonstrates the feasibility of hysteroscopic tubal occlusion of functionless hydrosalpinx in all cases with acceptable efficacy. It has the advantage of adding a valuable evaluation of the endometrial cavity prior to IVF/ICSI. It should be an option for treatment protocol in cases of functionless hydrosalpinges. Further large sample-sized studies are required to test its impact on the implantation rate and clinical outcome.

Implementation Evaluation of a Culturally Competent Eye Injury Prevention Program for Citrus Workers in a Florida Migrant Community

The Partnership for Citrus Worker Health (PCWH) is a coalition that connects academic institutions, public health agencies, industry and community-based organizations for implementation of an eye safety pilot project with citrus workers using the Camp Health Aide (CHA) model. This project was an implementation evaluation of an eye safety curriculum using modeling and peer-to-peer education among Mexican migrant citrus workers in a southwest Florida community to increase positive perceptions toward the use of safety eyewear and reduce occupational eye injuries. CHAs have been employed and trained in eye safety and health during harvesting seasons since 2004. Field observations, focus group interviews, and written questionnaires assessed program implementation and initial outcomes. There was an increase in positive perceptions toward use of safety eyewear between 2004 and 2005. Evaluation of training suggested ways to improve the curriculum. The modest literacy level of the CHAs necessitated some redesign of the curriculum and its implementation (e.g., introduction of and more reliance on use of training posters). PCWH benefited by extensive documentation of the training and supervision, a pilot project that demonstrated the potential effectiveness of CHAs, and having a well-defined target population of citrus workers (n = 427). Future research can rigorously test the effectiveness of CHAs in reducing eye injuries among citrus workers.

Antibody-Drug Conjugates with Uncleavable Linkers for the Treatment of Non-Hodgkin Lymphoma.

Antibody-drug conjugates (ADCs), potent cytotoxic drugs linked to antibodies via specialized chemical linkers, provide a means to increase the effectiveness of chemotherapy by targeting the drug to neoplastic cells while reducing side effects. Our research has focused on ADCs that contain the maytansinoid DM1, monomethylauristatin E (MMAE), or monomethylauristatin F (MMAF), all of which are potent inhibitors of microtubule polymerization. These drugs are attached to the antibody via several different linker formats (Table 1).Table 1Linker-drugLinker MechanismLinked viaDrug Cell PermeablePAB-vc-MC-MMAECleavage of peptide bondCysteineYesMC-MMAFUncleavableCysteineNoSPP-DM1Reduction of disulfide bondLysineYesMCC-DM1UncleavableLysineYesWe identified seven cell surface proteins expressed in non-Hodgkin lymphoma (NHL) with limited expression patterns in normal tissue (CD19, CD20, CD21, CD22, CD72, CD79, and CD180), as potential therapeutic targets for ADCs. ADCs directed to any of these seven targets are effective in xenograft models when containing linkers that can be cleaved in the tumor microenvironment or when internalized by tumor cells. However, ADCs with uncleavable linkers, i.e., linkers that are active when antibody is internalized and degraded within cells, are effective only when targeted to CD22 or CD79b. This suggests that these antigens provide the desired intracellular targeting of the cytotoxic drug. Further, we demonstrated that the uncleavable-linker anti-CD22 and anti-CD79b ADCs result in complete tumor regression with no recurrence in some xenograft models of NHL. In vitro experiments revealed that sensitivity of a given cell line to the ADCs anti-CD22-MCC-DM1 and anti-CD79b-MCC-DM1, correlated more with sensitivity of the cell line to the corresponding free drug, than to the amount of surface expression of the target or the amount of internalized ADC. We conducted pilot safety studies in rats (non-binding species) and cynomolgus monkeys (binding species). In rats, ADCs with uncleavable linkers caused less target-independent hepatic and hematologic toxicity than their counter parts with cleavable linkers, likely due to their decreased systemic release of free drug. Because of these results, we evaluated three ADCs with uncleavable linkers (anti-CD22-MCC-DM1, anti-cynomolgus monkey CD79b-MCC-DM1, and anti-CD22-MC-MMAF) at ∼30 mg/kg q3 weeks for 2 doses in cynomolgus monkeys. Normal B-cells were depleted in circulation and in tissue, particularly in germinal centers of lymphoid tissues; an expected pharmacological effect. Other findings included clinically tolerated and reversible elevations in liver enzymes (MCC-DM1 and MC-MMAF conjugates) and decreases in platelet counts (MCC-DM1 conjugates); and minimal to mild sciatic nerve degeneration (MCC-DM1 conjugates). The data demonstrate that anti-CD22 and anti-CD79b ADCs with uncleavable linkers have potent efficacy in xenograft models of NHL, favorable safety profiles in pilot animal studies, and suggest that for specific targets, uncleavable linkers provide a promising mechanism to improve margins of safety in humans.

Safety culture and hazard risk perception of Australian and New Zealand maritime pilots

A survey of the safety culture and hazard risk perception has been carried out involving 77 maritime pilots around Australia and New Zealand, representing more than the 20% of the maritime pilots in each country, in proportional geographic distribution. In 82% of the cases, interviews were carried out face-to-face, based on a questionnaire designed to be completed in less than 1 hour. Questions on 4 different aspects were asked, relating to each pilot's professional background, safety culture and perception of risks, navigation and pilotage hazards and their perception of the pilot's role. The responses indicated that there are issues with regulator identification, commercial pressures on pilots in some locations, incident reporting mechanisms and feedback, training opportunities and improved bridge resource management. In addition to reviewing comments made by pilots, the paper also presents recommendations for consideration in improving operational performance and safety of navigation in ports and regulated waterways. These recommendations can be used by regulators to improve their performance concerning pilotage matters and understand pilots’ concerns.

138: Intrauterine Ultrasound-Guided Radiofrequency Ablation of Fibroids: A Pilot Feasibility and Safety Study

138: Intrauterine Ultrasound-Guided Radiofrequency Ablation of Fibroids: A Pilot Feasibility and Safety Study

A pilot safety and tolerability study of a non-hormonal vaginal contraceptive ring

To report preliminary data on the Ovaprene® device, a medical grade silicone ring that releases ferrous gluconate, ascorbic acid, and glycine in spermiostatic concentrations (Poly-Med Inc, Clemson University), designed for over the counter non-hormonal contraception. An open label short term pilot tolerability study in 20 paid volunteers. Women using another contraceptive method were eligible if they met the other inclusion criteria. Women were instructed in proper insertion of the ring at the completion of their menses, with removal at the onset of their subsequent menses or 29 days, whichever was shorter. PAP, vaginal cultures, colposcopy and post-coital testing were performed at daily to weekly follow-up. To date, 10 women have enrolled and 5 completed one cycle use (total observations approximately 100). All rings were inserted properly and retained in place including post-coitus (range 1–29 days). Patient questionnaire revealed no pain, bleeding or discharge. Male partners detected the device (2 partners of the 10 women). Wet mounts were normal. Semi-quantitative cultures showed no significant changes in genital flora. Vaginal pH was ≤4.5 except post-coitus (range 1–4 hours) was 4.5–6.5. All epithelium was intact. Random ferrous gluconate vaginal fluid concentrations during normal usage averaged 1438 ± 1028.93 μg/g. Post-coital test revealed sperm (motile/total, mean ct/10hpf) vaginal pool 2/>20; cervical mucous 0/0. One patient had odor for 24 hours after intercourse resolving spontaneously. All participants were willing to use and recommend the product during sexual intercourse in the future, as reported on the follow-up acceptability questionnaire. The Ovaprene® device is well tolerated and acceptable to sexually active women. Preliminary post-coital test demonstrates theoretical contraceptive efficacy. Additional data will be presented.

Open-label safety and efficacy pilot trial of intraperitoneal bevacizumab as palliative treatment in refractory malignant ascites

9043 Background: One of the most burdensome complications of advanced cancer is the development of malignant ascites, impacting quality of life (QoL), gastrointestinal function, survival, and overall management costs. Recent studies have shown that malignant effusions arise in part from increased production and activity of vascular endothelial growth factors (VEGFs). VEGFs increase vascular permeability and establish an ideal environment for accumulation of malignant effusions. Methods: A pilot trial evaluating safety and efficacy of intraperitoneal administration of bevacizumab at 5 mg/kg monthly for as long as no disease progression or unacceptable toxicities are encountered. Patients with cytologically documented malignant ascites from any primary tumor refractory to standard therapy of diuretics, repeated paracentesis, and/or intraperitoneal chemotherapy were eligible. Patients were either off systemic therapy or had not had any changes in their systemic therapy for at least 4 weeks at the time of enrollment. Results: Between July 2006 and November 2006, 9 patients with refractory malignant ascites from colon cancer (3 pts), breast cancer (3 pts), uterine cancer (2 pts) and ovarian cancer (1 pt). Prior therapy included systemic chemotherapy and large volume paracentesis. Patients with ovarian and uterine cancer also received intraperitoneal cisplatin. All patients had rapid re-accumulation within 2 weeks of paracentesis before treatment. Patients were given intraperitoneal bevacizumab at 5 mg/kg monthly. Complications included grade I nausea in one patient, grade I abdominal pain in one patient, and partial small bowel obstruction in one patient that was managed conservatively. Malignant ascites resolved without reaccumulation or repeat paracentresis in 9/9 after a single intraperitoneal dose of bevacizumab over a median observation period of over two months. No objective tumor responses were observed. QoL assessment, pharmacokinetic studies of intraperitoneal Bevacizumab and pharmacodynamic studies on serum and ascites fluid VEGF are ongoing and will be reported. Conclusions: Intraperitoneal bevacizumab is a relatively safe and highly efficacious way to palliate the symptoms of refractory malignant ascites. No significant financial relationships to disclose.

404 A pilot safety and dose-finding trial of intravenous recombinant human relaxin (rhRlx) in compensated congestive heart failure

404 A pilot safety and dose-finding trial of intravenous recombinant human relaxin (rhRlx) in compensated congestive heart failure

Cardiovascular consequences of high-performance aircraft maneuvers: implications for effective countermeasures and laboratory-based simulations

The gravitational stress encountered by pilots of high-performance aircraft can cause dramatic shifts in blood volume and circulatory pressure, thus placing the cardiovascular system under significant stress, sometimes resulting in loss of consciousness due to cerebral under-perfusion. Since pilots experience both increased and decreased gravitational stress in high-risk environments, it is important not only to examine the cardiovascular effects of altered gravitational exposure, but also to create effective countermeasures that will increase pilot safety. In this review, we discuss the cardiovascular consequences of rapid changes in gravitational forces. We also examine the effectiveness of the countermeasures that have been developed to combat gravity-induced loss of consciousness. Finally, we examine those current laboratory-based techniques that simulate hyper-gravity and the "push-pull effect"; making it possible to investigate the cardiovascular mechanisms responsible for maintaining cerebral perfusion and consciousness.

Safety and feasibility of acute percutaneous septal sinus shortening: First‐in‐human experience

Multiple percutaneous therapies for the treatment of functional and ischemic mitral regurgitation (FMR/IMR) are under development. We previously reported a novel percutaneous technique, the percutaneous septal sinus shortening [PS(3)] System which was effective in ameliorating FMR in an animal model. We herein report results from the first-in-human safety and feasibility pilot study involving the PS(3) System. The primary objective of this first-in-human study was to evaluate the safety and feasibility of acute percutaneous septal-lateral shortening by using the PS(3) System in patients immediately prior to clinically-indicated surgical mitral valve repair. Two patients were enrolled. Patient One had severe aortic insufficiency with moderate functional mitral regurgitation. The PS(3) System reduced the MR grade from 2+ to 1+ with a decrease in the mean septal-lateral systolic (SLS) dimension from 38 to 27 mm (29% reduction). Patient Two had severe ischemic mitral regurgitation in the setting of severe multi-vessel disease and prior infero-posterior infarct. MR grade was reduced from 3+ to 1+ with a decrease in the mean SLS dimension from 36 to 25mm (31% reduction). There were no procedural complications and both patients proceeded to pre-planned cardiac surgery, where the devices were explanted under direct visualization. The PS(3) System has been safely translated from the preclinical setting to first-in-human implantation. Both patients studied experienced a reduction in MR after device implantation, with significant SLS shortening. Further clinical trials will be needed to assess long-term efficacy and durability.

Results and recommendations from the helicopter EMS pilot safety survey 2005

Introduction In 2005, the Pilot Safety Study Group (PSSG), consisting of members of the Association of Air Medical Services Research Committee, wrote, distributed, and analyzed a survey of helicopter pilots regarding their knowledge, attitude, and perspectives on safety in the field of air medical transport. Methods The Pilot Safety Survey 2005 (PSS2005) was based on another survey—one that was sponsored by Helicopter Association International (HAI) and National EMS Pilots Association (NEMSPA) and administered to pilots in 2001. The PSS2005 pared questions down so that the survey could be completed in 15 minutes on the internet, and the answers were organized in a manner to simplify analysis. An electronic link to the survey was distributed in a non-randomized fashion to HEMS pilots using the mailing lists of various operators and HEMS programs. Questions were clustered into eight groupings of safety, with a majority of responses being categorical, lending themselves to cross-tabulations. Results The information gathered indicated that Helicopter EMS (HEMS) pilots are very experienced, with the average pilot logging 6,625 flight hours. Collectively, they took responsibility for HEMS accidents; with 92% of total respondents citing “pushing weather minimums” and 82% citing “pilot decision making” as the main reasons for crashes. Crew resource management (CRM) was well appreciated by the pilots; there appeared to be a positive correlation with programs that offer their employees CRM and the pilots' general perspective on safety. The survey was also clear that amongst 40% of the respondents, mission-oriented training needs improvement, and 74% responded that more realistic training in flight simulators would improve safety overall. Finally, 57% of the pilots both desired night vision goggles or devices (NVG/NVD) and believed that their usage would improve safety in the field of air medical transport (55% vs 45%, P = .0025). Conclusions Although the recommendations from the PSS2005 are lacking in definite evidence for a decrease in HEMS crashes, we consider the direct input from pilots as critical in the absolute elimination of crashes in Helicopter EMS (Vision Zero). Pilots are, after all, the very ones held responsible for HEMS crashes. Based on these findings, the PSSG hopes that the HEMS community will incorporate the following recommendations into their standard practices. We recommend that all HEMS operators have annual and regular CRM training. We recommend that all HEMS pilots have annual and regular training in realistic flight simulators. Finally, we recommend that all HEMS aircraft be in possession of NVGs, and if this is not possible (eg, light pollution from a highly urbanized region or cost–benefit issues), then to have annual and regular mission-oriented nighttime training.

Master training in Agribusiness and Rural Development at the University of Zagreb

The title of the proposed JEP project is: “Agribusiness Higher EducAtion Development” with the acronym AHEAD. This curriculum development project – in case of acceptance – will last for three years, from July 2005 and June 2008. The primary project site is the University of Zagreb, Croatia; the contractor and the co-ordinator institution is the University of Debrecen, Hungary. In the consortium, 3 further European universities (University of Hohenheim, Wageningen University and Scottish Agricultural College) will participate, from the Croatian side the Ministry of Agriculture, Forestry and Water Management and an additional 7 Croatian institutes will also be involved. The total number of members in the consortium is 13. The main objective of the AHEAD project is to establish new BSc and MSc programmes in Croatia at two Faculties of the University of Zagreb. These are as follows: Agricultural and Rural Development, Food Safety and Quality Management and pilot MSc training in Agribusiness and Rural Development. These are preceded by faculty retraining programmes in food safety and quality management, as well as agribusiness and commerce within the framework of a MBA programme accredited by the International MBA Network. The professional content of the project is a modernised curriculum and training palette that would be available by the end of this project at the University of Zagreb, serving not only the higher education of the country, but the demand of the Croatian national economy as well, in line with the basic principles of the European Union.

Pilot safety survey 2005: What pilots are saying

Pilot safety survey 2005: What pilots are saying

Aspiration curette for anterior capsule polishing: Laboratory and clinical evaluation

Capsular fibrosis or fibrotic after-cataract results from transdifferation of anterior lens epithelial cells into myofibroblasts with consequent contraction and collagen deposition. To avoid possible complications, an instrument was designed for efficient and safe polishing of the anterior capsule leaf. The curette features an upward-facing slit with sharp-edged flanks and rounded turning points and an optional bypass hole. It is introduced through 3 equidistant 20-gauge paracentesis openings. In a laboratory test, vacuum rise time and vacuum levels under occlusion and the effect of adding a bypass hole were investigated for various pump settings. In a clinical pilot series, efficiency and safety of various designs were tested and vacuum and flow settings titrated for optimum performance; the clinical effect was determined in prospectively randomized bilateral studies. Optimum pump settings were 5 mL/min and 10 mm Hg with the nonventing curette and 7 to 10 mL/min and 50 mm Hg with the venting curette. A bypass hole delayed the vacuum rise and reduced the mean vacuum level on full occlusion. It also allowed fine-tuning the vacuum at the slit by varying the flow rate with the foot pedal. Anterior capsule polishing significantly decreased capsule fibrosis and is recommended when posterior capsulorhexis and optic buttonholing are combined.

Trials in Sickle Cell Disease

Children with sickle cell disease are at risk of developing neurologic complications, including stroke, transient ischemic attack, seizures, coma, and a progressive reduction in cognitive function. Transcranial Doppler ultrasound, magnetic resonance imaging, and overnight pulse oximetry appear to predict, making prevention an achievable goal so that there is now a focus on randomized controlled trials. The Stroke Prevention Trial in Sickle Cell Anemia (STOP) reported a reduction in the number of overt clinical strokes experienced by those children with critically high transcranial Doppler velocities (>200 centimeters per second) who were chronically transfused. Two additional Phase III studies and two pilot trials have been funded. STOP II focused on whether it is safe to discontinue blood in prophylactically transfused children when their velocities had remained normal for at least 30 months. The Silent Infarct Transfusion trial is designed to determine whether children with sickle cell anemia and silent cerebral infarcts, approximately 20% of the population, will have a decrease in the progressive neurologic complications after receiving regular blood transfusion therapy. Pilot safety and feasibility trials of low-dose aspirin and overnight respiratory support are also beginning. The collaboration provides an infrastructure for future clinical trials in this vulnerable group of children.

Extracorporeal photochemotherapy: a safety and tolerability pilot study with preliminary efficacy results in refractory relapsing-remitting multiple sclerosis

Extracorporeal photochemotherapy (ECP) is an immunomodulating procedure consisting of autologous reinfusion of peripheral blood mononuclear cells (PBMC) after direct exposure to 8-methoxy-psoralen and UV-A. It has been described as a successful treatment for different T-cell-mediated diseases and preliminary results suggest that ECP might be effective in the treatment of relapsing-remitting multiple sclerosis, but does not significantly alter the course of the progressive form of MS. In this study, we report the safety data and some preliminary efficacy evidence obtained using ECP in the treatment of five patients with refractory relapsing-remitting (RR) MS: in most cases ECP induced a reduction in the relapse rate and an EDSS stabilisation, with an apparent general MRI stabilisation. In conclusion, our results confirm ECP safety and tolerability and suggest that this treatment might be useful as a therapeutic alternative in the subgroup of RRMS patients not responsive to or not eligible for traditional immunomodulating or immunosuppressive treatments.