Results: All patients had successful disimpaction over 3 days (mean 6 cups of stool in total) and then continued with low dose of medication and TES therapy. All started with <3 bowel actions/week. After 8–12 weeks of TES, 32/33 (97%) increased to >3

BA/wk with 29 /33 Selleck Hydroxychloroquine (88%) having 7 BA/wk. Median stool consistency improved from BSS score of 2 (range: 1–7) to 4 (4–5) (p < 0.0001). Median stool output improved from 1 (0–2) to 7 (2–10) cups/wk (p < 0.0001). Soiling episodes decreased from 5 (0–7) to 0 (0–4) episodes/wk (p < 0.0001). Patients were weaned off laxatives during TES, and off TES after 3 months and continued with daily defecation. Conclusions: We have previously shown that TES added onto existing treatment increases defecation to >3BA/wk in half of the patients with STC over 2–3 months (Yik 2012). The addition of disimpaction with oral laxatives and education on diet and toileting prior to TES therapy resulted in >3 BA/wk in 97% of patients with 88% having daily bowel motions. Improvement occurred in more patients, was bigger improvement and was more rapid than with TES alone. TES is a non invasive treatment 1. Yee Ian Yik, Khairul A Ismail, John M Hutson, Bridget R

Southwell. 2012. Home transcutaneous electrical stimulation to treat children with slow-transit constipation. J Pediatr Surg 47(6): 1285–1290. 2. Jordan-Ely J, Hutson JM, Southwell BR. EPZ6438 Lifestyle Approach: Holistic Management. In: Constipation: Current & Emerging Treatments. Future Medicine 2013 (In press). J JORDAN-ELY,1,2 K DOBSON,1 JM HUTSON,1,2,3 BR SOUTHWELL1,3 1Murdoch childrens Research Institute, Parkville, Australia, 2Dept. Urology, Royal Childrens hospital, Parkville,

Australia, 3Dept. Paediatrics University of Melbourne, Parkville, Australia Introduction: polyethylene glycol (PEG) is an oral stool softener that produces disimpaction in 97% patients. However because of the large selleck screening library volume (2 litres) that needs to be taken, many patients have difficulty completing treatment. We have developed a program of patient education and engagement (called MOTIVATE) to enable compliance and obtain the highest efficacy. The aim of the study was to review outcomes of oral bowel disimpaction with PEG administered in a nurse-led clinic using the MOTIVATE method. Materials and methods: A retrospective clinical audit of 33 patients (2–17 years, 17 male) with chronic constipation referred to a surgeon at a tertiary Childrens hospital. Patients and carers were provided information on Diet, Education, Laxative and Disimpaction (DELD) method during two × 30 min sessions. An advanced practice nurse demonstrated how to take the PEG+E (Movicol) combined with Sodium Picosulphate (Dulcolax SP). The solution was mixed with 125 ml of water/sachet. The mixture was taken spread out across the morning at a rate of 125 ml/hour. To make drinking easier and fun, 125 ml was divided into 6 shot glasses and an equal volume of juice added.

Of 242 patients randomized, 216 were included in the intention-to-treat efficacy analysis. In the SPr group, 39.6% of subjects experienced 2-hour headache-free response (primary outcome), which was significantly more effective than SP treatment (26.3%, odds ratio: 1.83, 95% confidence interval: 1.03–3.26, P = .038).

Significantly more patients receiving SPr treatment (62.2%) had headache improvement compared with SP treatment (37.2%) at 2 hours (odds ratio: 2.77, PF-562271 purchase 95% confidence interval: 1.60-4.81, P < .001). A similar pattern of between-group differences was observed for 4-hour headache-free response (P = .006) and headache improvement response (P = .003). The incidence of headache recurrence within 2-48 hours after treatment was lowest in the SPr group (15.0%) compared with SP group (26.6%, P = .041). The only significant

drug-related adverse events reported in ≥15% of patients in any treatment group were somnolence (32.2% and 7% in the SPr and SP groups, respectively, P < .001), extrapyramidal symptoms (4.3% and 0%, P = .05), and nausea (1% and 8%, P = .03). This is the first prospective clinical trial to demonstrate that multimechanism therapy for migraine, combining a triptan and an antiemetic agent, is well tolerated and offers improved clinical benefits compared with monotherapy. PF-6463922 manufacturer Migraine is a chronic, multifactorial, and debilitating neurological disorder characterized by recurrent moderate to severe attacks of headache and autonomic dysfunction.[1] It is commonly accompanied by several symptoms such as nausea, vomiting, phonophobia, photophobia, and aggravation by exertion.[2] According to previous population estimates, the current

global prevalence of headache and migraine are 47% and 10%, respectively.[3] Migraine imposes considerable social and economic burdens on individual headache sufferers and society.[4] The severity and extent of symptoms result in considerably impaired social function, increased utilization of medical services, and reduced health-related quality of life.[5, 6] Migraine has historically been an underestimated and undertreated disorder.[3] check details Furthermore, approximately half of migraine patients discontinue looking for treatment for their headaches, partly due to dissatisfaction with therapy. Indeed, public surveys revealed that headache sufferers are among the most dissatisfied patients.[7] Therefore, efficient management should include establishing logical expectations, patient assurance, and appropriate medical treatment and instruction.[8] Moreover, successful treatment of migraine attacks could benefit migraineurs by reducing their disability and the need for health care resources, and improving economic productivity.

Also, the selfish genetic interests of interacting organisms tend to be aligned only insofar as those individuals are related (Hamilton, 1964; Mock & Parker, 1997), and pairs of individuals in a nuclear family differ dramatically

in their coefficients of genetic relatedness (r): a mother and her offspring normally share half their genes (r = 0.5) as do full sibs in a multi-birth litter; but half-sib progeny share only one-quarter of their genes (r = 0.25), and a sire and dam typically are unrelated (r = 0.0). For these and other reasons, each nuclear family is not simply a serene setting for harmonious interactions, but rather it can be an evolutionary minefield of oft-competing genetic fitness interests, both inter- and intragenerational (Trivers, 1972, 1974; Hausfater & Hrdy, 1984; Parmigiani selleck chemicals llc and Vom Saal, 1994; Hudson & Trillmich, 2008). Furthermore, many of these conflicts play out forcefully within the mammalian womb. Thus, pregnancy becomes an evolutionary theatre for intergenerational conflict over parental resources – each offspring is under selection to seek as many maternal resources as possible (limited

only by any negative effects on its inclusive fitness that such demands impose on copies of its genes carried by its kin), whereas a dam can be expected to resist excessive demands by the fetus. The net result of each such evolutionary ‘tug-of-war’ (Moore & Haig, 1991) between mother and child is some ontogenetic balance in which each offspring must settle for fewer maternal resources than it ideally might wish and a mother surrenders more resources than she otherwise might prefer. But by evolutionary MK-8669 cell line reckoning, any such maternal–fetal compromise during or after a pregnancy is less the result of a harmonious mutualism than it is an outcome of conflict mediation (Haig, 1993, 1999, 2010; Nesse & Williams, 1994). Of course, maternal–offspring relations entail elements of cooperation as well as conflict;

these two categories of interaction need not always be interpreted as mutually exclusive (Strassmann et al., 2011). Selective pressures that pregnancy promotes sometimes have led to outcomes that catch researchers totally off-guard. One such phenomenon is genetic imprinting: a situation in which a gene is expressed in progeny when inherited from one parent but find more not from the other (Solter, 1988). In such cases, a gene can have very different effects on offspring (and therefore on the course of a pregnancy) depending on whether it was transmitted via the dam (egg) or sire (sperm). Genetic imprinting in animals appears to be confined mostly to viviparous mammals, but the phenomenon also is common in plants (Feil & Berger, 2007). In recent years, scientists have discovered imprinted genes in many marsupial and placental mammals, including Homo sapiens, where imprinting has been documented at approximately 100 loci to date.

29 [95% CI: 0.14–0.60] for the high affinity tertile, P = 0.002), the C2 domain-restricted analysis indicated an inverse Selleck BMN-673 correlation (OR = 3.56 [1.10–11.52], P = 0.03).

Our data validate the importance of the affinity of FVIII peptides for HLA alleles to the immunogenicity of therapeutic FVIII in patients with missense mutations. “
“Summary.  Haemarthroses (intra-articular haemorrhages) are a frequent finding typically observed in patients with haemophilia. Diagnosis and treatment of these bleeding episodes must be delivered as early as possible. Additionally, treatment should ideally be administered intensively (enhanced on-demand treatment) until the resolution of symptoms. Joint aspiration plays an important role in acute and profuse haemarthroses as the presence of blood in the joint leads to chondrocyte apoptosis and chronic synovitis, which will eventually result in joint degeneration (haemophilic arthropathy). Ultrasonography (US) is an appropriate diagnostic technique to assess the evolution of acute haemarthrosis in haemophilia, although magnetic resonance imaging remains the gold standard as far as imaging techniques are concerned. Some patients experience subclinical haemarthroses, which eventually tend to result in some

degree of arthropathy, especially in the ankles. Nowadays, the most effective way of protecting these patients is primary prophylaxis, which in practice changes severe haemophilia into moderate haemophilia, preventing or at least minimizing the occurrence MLN0128 of haemarthrosis. If primary prophylaxis is, for whatever reason not an option, secondary prophylaxis and find more enhanced on demand treatment should be considered. Two alternatives are available for inhibitor patients: (i) control of haemostasis using by-passing agents (rFVIIa or aPCCs) either as enhanced on demand treatment or secondary

prophylaxis, as appropriate, following the same basic principles used for non-inhibitor patients and (ii) immune tolerance induction (ITI) to eradicate the inhibitor. “
“A number of observations suggest that severe factor IX deficiency (<1%) may be less clinically severe than the corresponding factor VIII deficiency: (i) Less factor consumption. There is evidence that patients with haemophilia B (HB) consume yearly less FIX for replacement therapy than patients with haemophilia (HA). Patient registries and data from various sources indicate that regular prophylaxis is implemented less frequently in HB. (ii) Less severe gene mutations. At variance with HA, missense gene mutations are prevalent in severe HB, supporting the view that some FIX may be produced in these patients, albeit not measurable in patient plasma by means of the relatively insensitive available assays. (iii) Less severe clinical symptoms.

The aim of this study is to determine listing practices for morbidly obese

patients in United States (U.S.) liver transplant centers. Methods: A 19 item survey was created to assess liver transplant evaluation and listing practices for morbidly obese patients. All U.S. adult liver transplant medical and surgical directors were contacted by email with a cover letter describing the study and an internet link to the SurveyMonkey® website. A few questions had a free-text section which allowed for comment. Five follow-up emails were sent to encourage participation. Results: A total of 187 surveys were emailed with responses received from 46 physicians (24.7% response rate). The responding cohort PD98059 manufacturer consisted of 29 (63%) medical directors and 17 (37%) surgical directors, including respondents from all United Network Organ Sharing (UNOS) regions, though regions 4 and 6 had the fewest respondents (n=2). The majority of respondents reported treating patients at an academic medical center (73.3%) and performing more than 50 liver transplants a year (60.8%). A policy on evaluation and listing of obese patients

was present at 70.5% of institutions with the majority (54.5%) reporting their BMI cut off for transplant was 40 but a range of 35 to unlimited was noted. The majority (61.4%) of KPT-330 mouse respondents agreed that there has been an increase in the number of obese patients they have listed for liver transplant, however 75% of

respondents’ reported learn more that patients with high BMI were less likely to be evaluated for transplantation. With regards to complications in obese patients, 65.9% of respondents reported experiencing an increased complication rate, with the most frequently cited complications being poor wound healing and increased infection rates. Despite the reported increased complication rate, only 34.1% reported they had experienced worse survival rates with obese patients. Conclusions: The majority of medical and surgical liver transplant directors have a strong appreciation of the possible morbidity risks associated with morbidly obese patients post-transplant and have policies in effect to minimize these risks. This is of specific concern due to the need to provide more high quality and cost effective transplant care in the current healthcare climate. More data examining morbidly obese cirrhotic patient outcomes perioper-atively, stratified by other co-morbidities, is needed. Disclosures: Jonathan M. Fenkel – Consulting: Gilead Pharmaceuticals, Janssen Therapeutics The following people have nothing to disclose: Dina Halegoua-De Marzio, She-Yan Wong, Cataldo Doria, David A. Sass Background: Racial/ethnic disparities in liver transplantation (LT) are well established. African Americans (AAs) are referred for LT at lower rates, and there is significantly lower post-LT survival among AAs compared to other groups.

The

study was a prospective investigation of the changes in immunoregulatory markers in the blood, bone marrow, and liver allograft in recipients converted from TAC monotherapy to SRL monotherapy for clinical indications (e.g., TAC toxicity). Inclusion criteria were as follows: age ≥18 years; ≥6 months post-LT; TAC monotherapy ≥1 month before SRL monotherapy conversion for nephrotoxicity (glomular filtration rate [GFR] 30-60 cc/min by modified diet in renal disease [MDRD]) or other indication; ≥6 months without a rejection episode; Pexidartinib no lymphocyte depletion therapy for ≥1 year; normal liver-function tests; and no rejection or fibrosis on preconversion liver biopsy. Exclusion criteria were as follows: previous liver or multiorgan transplant; previous immune or viral liver disease unless hepatitis C virus (HCV) RNA was undetectable; proteinuria (≥0.5 g/day); estimated glomerular filtration rate (eGFR) ≤30 cc/min; ≥2 rejections

post-LT; history of hepatic artery thrombosis; hematological abnormalities or severe hypertriglyceridemia; active infection or malignancy; and inadequacy for follow-up. All patients signed informed consent and were followed for 7 months after SRL conversion. The protocol conformed to the Declaration of Helsinki guidelines and was approved by the Northwestern Institutional Everolimus in vivo Review Board (Northwestern University Feinberg School of Medicine, Chicago, IL). History and physical exams, complete blood counts, comprehensive metabolic panels, fasting lipids, hemoglobin A1C tests (HgA1C), and spot urine protein:creatinine ratios were performed before and 3 and 6 months after conversion. Bone marrow aspirations and percutaneous liver biopsies were performed once before and 6 months after conversion. For conversion, SRL at 2 or 3 mg (< or ≥100 kg body weight) daily was initiated with subsequent weekly SRL trough-level monitoring. When these reached ≥5 ng/mL, TAC was discontinued followed by weekly laboratory tests and SRL trough levels (goal, 5-8 ng/mL) for 1 month,

then monthly. Prospective liver- and renal-function tests, lipid levels, urine protein:creatinine selleck chemicals llc ratios, and any new SRL toxicities were recorded. Treg immunophenotyping (twice before conversion and 3, 4, 6, and 7 months after conversion): Peripheral blood mononuclear cells (PBMCs) were isolated from heparinized samples on Ficoll-Hypaque gradients. Tregs were enumerated utilizing extracellular immunofluorescent staining with CD3-FITC (fluorescein isothiocyanate), CD4-PerCP (peridinin-chlorophyll protein complex), CD8-PerCP, CD25-APC, and CD127-FITC (BD Biosciences, San Diego, CA). After fixation and permeabilization, the cells were washed and incubated with anti-human FOXP3-PE (phycoerythrin) or rat immunoglobulin G2a-PE isotype control (eBioscience, San Diego, CA) (21, 22).

His vital signs were within normal limit. He buy Small molecule library had diffuse abdominal tenderness, especially in left upper quadrant and guarding. The laboratory findings were not significant. The CT showed 15 cm length intestinal wall

edematous enlargement at jejunum and high density area at mesentery around jejunum and ascites at Douglas cavum. He was radiologically diagnosed with small intestinal anisakiasis. It was resolved spontaneously in a few days. Conclusion: Discussion: Acute gastric anisakiasis can be easily diagnosed by the endoscopic visualization of Anisakis larvae along with mucosal edema, erythema, hemorrhage, and/or an ulcer. However, small intestinal anisakiasis 3 MA is difficult to diagnose because we cannot endoscope it easily. The CT scan typically showed severe intestinal submucosal edema with ascites. The small intestinal anisakisis should be considered by the food history and the typical CT finding. If strongly suspected, small intestinal anisakaisis can be treated without surgery because the larvae will die within a few days and the symptoms will subside soon. Key Word(s): 1. Anisakiasis Presenting Author: OSAMU OGAWA Additional Authors: YUGO SUZUKI, AKIRA MATSUI, TOSHIFUMI MITANI, SHU HOTEYA, MITSURU

KAISE Corresponding Author: OSAMU OGAWA Affiliations: Toranomon Hospital, Toranomon Hospital, Toranomon Hospital, Toranomon Hospital, Toranomon Hospital Objective: Gastric adenocarcinoma of fundic grand type (GAFG) is neoplastic lesion mainly composed of highly differentiated columnar cells mimicking the fundic gland cells with nuclear atypia. It has been reported as a new, rare variant of gastric adenocarcinoma. Therefore, its endoscopic features are uncertain. The aim of the current study was to evaluate the endoscopic features of GAFG. Methods: From October 2012 to March 2013, three

consecutive patients with GAFG resected by endoscopic submucosal dissection (ESD) in our hospital were enrolled in this retrospective study. These specimens resected by ESD revealed well-differentiated adenocarcinoma mimicking fundic gland cells, which were positive for pepsinogen-1 selleck inhibitor (a marker of chief cells) and MUC6 (a marker of fundic gland cells). These findings were consistent with GAFG. To evaluate the endoscopic features of GAFG, they were examined for their location, background mucosa, shape, color, and size. Results: All three GAFGs were in the upper part of the stomach. In the background mucosa, all they had normal fundic gland mucosa without atrophic change. And all they had whitish submucosal tumor shape with dilated branching vessel, ranging in size from 5.0 to 6.0 mm (mean, 5.1 mm). Conclusion: Precise understanding of these endoscopic features must enhance efficacious detection of GAFG in endoscopic surveillance. Key Word(s): 1.

Results: Chronic hepatitis B and cirrhosis due to hepatitis B virus were responsible for 57,380 DALYs in the country (30.3 per 100,000 inhabitants), with 41,262 DALYs for men and 16,118 DALYs for women. DALYs were mainly

caused from YLL rather than YLD (47,015 or 24.8/100,000 vs 10,365 or 5.5/100,000). There were 207,747 DALYs (109.6/100,000) attributable to chronic hepatitis C and cirrhosis due to hepatitis C virus, of which 137,922 were YLL (72.7/100,000) and 69,825 (36.8/100,000) were YLD. Male preponderance was also observed (73.9% of DALYs). Cirrhosis due to alcohol or other causes led to 536,169 DALYs, accounting for 1.4% of total disease burden in the country and representing the 11th cause of DALY in men. For this condition, there were 418,272 YLL (341,140 in men and 77,132 in women) and 117,897 YLD (97,965 in men and 19,931 in women). When analyzing age distribution, the highest DALYs’ rates were found at ages 60-69 in chronic hepatitis

C646 chemical structure B and C and at ages 45-59 in cirrhosis due to alcohol or other causes, Conclusion: Chronic hepatitis and cirrhosis are relevant health problems in Brazil, especially in men. Although chronic hepatitis C had a high impact on DALY, alcohol related liver disease was its main related cause. The mortality component of DALY was of greater magnitude in the burden of all these conditions. Data shown are crucial for planning public health Selleck MLN0128 policies toward these diseases. Disclosures: The following people have nothing to disclose: Juliana R. de Carvalho, Flávia B. Portugal, Luísa S. Flor, Mônica R. Campos, Renata M. Perez, Cristiane Ville-la-Nogueira, Joyce Mendes A. Schramm BACKGROUND: Due to high costs and numbers of candidates for interferon-free HCV drug regimens, non-invasive tests of fibrosis have been proposed as a tool for prioritizing access to treatment. APRI and FIB-4 scores are readily available and reliably predict fibrosis (Ann Intern Med. 2013;158:807-820). The purpose of this study is to determine the consequences of prioritizing treatment to patients with Low, Intermediate (Int), and High score categories, both for predication of actual fibro-sis and

short term all-cause and liver-related events. METHODS: Retrospective study of 396 patients with baseline APRI, find more FIB-4, and liver biopsy data who received antiviral treatment. Patients were followed for a median of 9.58 (SD 3.62) years for response to antiviral therapy, all cause and liver-related events (liver transplant, liver death, HCC). The risk of death and liver-related events in each FIB-4 and APRI category was examined using Cox regression analysis. RESULTS: Baseline noninvasive testing categorized patients as APRI Low/Int/High n=75/185/136, and FIB-4 Low/Int/High n= 119/163/112. The presence of significant (stage 3-4) fibrosis in biopsies at baseline was 80-82%, 35-42%, and 19-20% in the High, Int, and Low categories, respectively. SVR reduced risk of liver death by 2.5-3.4 fold, 6.9-7.6 fold, and 11.0-4.

Indeed, in the recent trial comparing sunitinib versus sorafenib, survival under sorafenib was significantly better, whereas PFS was not different.45 This failure of PFS to reflect survival has also recently been shown for breast cancer treated with

bevacizumab.46 The same consideration may be applied to the use of recurrence-free survival (RFS) in treatment to prevent recurrence after resection or ablation. There is no proof of correlation between RFS and survival, and differences in RFS may be the result of its composite nature that implies a mix of death caused by cancer and deaths resulting from the progressive liver disease.1 As a result, regulatory agencies base their decisions for registration on a positive result in survival, whereas the other endpoints (e.g., RR, TTP, TTUP, and PFS) are mere suggestions that may prove correct in predicting survival benefit. In summary, imaging techniques selleck chemical are a central tool in clinical decision making. Any team willing to provide state-of-the-art clinical care and engage in research

should secure the active involvement of expert radiologists. If such commitment is not in place, quality of care will be suboptimal, and the advances provided by technology will not be properly implemented for the benefit of the patients and the cost-effective use of the expensive resources needed in cancer management. “
“Interleukin (IL)-33, a member of the IL-1 cytokine family, positively correlates with acute hepatitis and chronic liver failure in mice and humans. IL-33 is expressed in

buy Opaganib hepatocytes and is regulated by natural killer T (NKT) cells selleck inhibitor during concanavalin A (ConA)-induced acute liver injury. Here, we investigated the molecular mechanisms underlying the expression of IL-33 during acute hepatitis. The expression of IL-33 and its regulation by death receptor pathways was investigated after the induction of ConA-acute hepatitis in wildtype (WT), perforin−/−, tumor necrosis factor related apoptosis inducing ligand (TRAIL)−/−, and NKT cell-deficient (CD1d−/−) mice. In addition, we used a model of acute liver injury by administering Jo2/Fas-antibody or D-galactosamine-tumor necrosis factor alpha (TNFα) in WT mice. Finally, the effect of TRAIL on IL-33 expression was assessed in primary cultured murine hepatocytes. We show that IL-33 expression in hepatocytes is partially controlled by perforin during acute liver injury, but not by TNFα or Fas ligand (FasL). Interestingly, the expression of IL-33 in hepatocytes is blocked during ConA-acute hepatitis in TRAIL-deficient mice compared to WT mice. In contrast, administration of recombinant murine TRAIL associated with ConA-priming in CD1d-deficient mice or in vitro stimulation of murine hepatocytes by TRAIL but not by TNFα or Jo2 induced IL-33 expression in hepatocytes. The IL-33-deficient mice exhibited more severe ConA liver injury than WT controls, suggesting a protective effect of IL-33 in ConA-hepatitis.

Despite the financial barriers that haemophilia A patients encountered during the economic downturn,

only a portion of patients with difficulties paying OOP treatment costs received patient assistance. These findings suggested underutilization of patient assistance programmes and a need for patient and HCP-focused education and outreach regarding available resources for patients with financial constraints. Similarly, a web-based survey by Buell et al. involving 100 community oncology practices, estimated that about 10–11% of their patients were uninsured or underinsured and recommended that they take advantage of patient assistance programmes [25]. Assistance programmes provide access for patients who have financial difficulty to receive optimal care. Building awareness of INCB024360 cell line these programmes within the HCP and patient community may improve utilization. Although patients Romidepsin cost have indicated that they made treatment-related decisions due to insurance coverage status and financial constraints, inadequate patient access to care and insurance coverage limitations similarly interfered with HCPs’ ability to treat haemophilia patients. For example, treatment modifications made by HCPs participating

in this survey included delaying elective surgery (25%) and initiating prophylaxis treatment (15%). However, the per cent of HCPs making suboptimal treatment decisions has to be interpreted with caution. Unlike the patient survey, HCPs considered their total haemophilia A patient population when answering the questions. The answers only indicated that HCPs would consider making such modifications for at least one patient, likely not all patients, facing financial difficulties. Treatment strategies still varied among haemophilia A patients. Hence, the actual per cent of patients making suboptimal changes due to physician recommendations may be much smaller. Nevertheless, these treatment modifications are not in patients’ best interest and considered to be a deviation from the treatment guideline set by Medical and Scientific check details Advisory Council (MASAC), which recommends prophylaxis as the optimal therapy for individuals with haemophilia

A [4]. Aside from considering treatment modifications during the economic downturn, this study sought to understand participants’ familiarity with, and thoughts on the perceived impact of, health care reform on treatment decision-making. Unlike the economic downturn, health care reform was perceived by patients and HCPs to be positive for haemophilia A treatment by expanding access to health insurance coverage. Participants anticipated that the provisions from the enactment of health care reform law would enable more optimal treatment options for previously financially constrained patients and more prescribing flexibility for HCPs. The elimination of the lifetime caps was perceived by patients and HCPs as the most relevant health care reform provision for haemophilia A treatment.