Browse

1 - 10 of 330 items :

  • Clinical Medicine, other x
  • Pharmacology x
  • Clinical Medicine x
Clear All

Abstract

Haemophilia patients with inhibitors suffer from increased morbidity and mortality due to the ineffectiveness of factor VIII replacement. Pseudotumors are rare but dangerous complications in these patients, and nasal pseudotumors are even rarer. Here, we present the case of a young child with severe haemophilia A with high titre inhibitors who developed a nasal pseudotumor. When immune tolerance therapy was not possible due to financial constraints, he was treated with FEIBA prophylaxis and rituximab. The pseudotumor was managed with surgical excision. We conclude that epistaxis in haemophiliacs can be due to an underlying nasal pseudotumor, and highlight the use of rituximab for the eradication of inhibitors.

Abstract

Background

Disease-specific camps present one means of helping children overcome the challenges associated with chronic conditions and improving clinical and psychosocial outcomes. For more than 50 years, bleeding disorders camps (BDCs) in the United States (US) have been promoting independence, self-care, and leadership skills in children with bleeding disorders, all while fostering camaraderie in a secure and safe environment. However, little is known about how BDCs are organised, administered, funded, staffed, or how staff are compensated.

Aim

This article aims to describe the attributes of BDCs that service the US bleeding disorders community, and to compare and contrast these attributes to identify gaps in the BDC system and areas for improvement.

Methods

The National Hemophilia Foundation (NHF), in collaboration with several members of its Nursing Working Group and Physical Therapy Working Group, developed a survey that was distributed to BDC administrators (CAs) and health care providers (HCPs).

Results

A total of 101 HCPs and 20 CAs completed the survey. Findings indicated that BDCs are an informal extension of both the HTCs and NHF chapters, reaffirming that camps play a crucial role in the overall care of bleeding disorders. In general, diminishing financial resources threaten the existence of BDCs. Although there are BDC guidelines for formal staff training and specific interventions delivered to camp participants, adherence is variable. Other gaps included minimal self-infusion education follow-up with no documentation on effect or benefit of infusion education provided at camp.

Conclusion

Addressing the gaps identified by this survey and documenting resultant data supporting the value of BDCs will facilitate their continued sustainability in light of increasingly limited funding.

Abstract

Background

Recent treatment option advances in haemophilia care have contributed to a discourse of ‘normality’ around the condition, in which people with haemophilia (PwH) are increasingly expected to live ‘normal’ lives unburdened by their condition.

Aim

The aim of this article is to explore notions of ‘normality’ in the experience of haemophilia across generations. This is one of the main themes identified in a large-scale ethnographic study of the everyday life of PwH, a broad qualitative investigation of beliefs and experiences related to condition, treatment, and personal ways of managing the condition.

Methods

The study used ethnographic research methods. Five haemophilia experts helped frame the research design by contributing historical and disease area context prior to the initiation of field research. PwH were recruited through patient organisations in five European countries (Italy, Germany, Spain, UK and Ireland). During field research, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of PwH interactions with friends, family, and health care professionals (HCPs). They also conducted on-site observation at haemophilia treatment centres (HTCs) and HCP interviews. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenges mapping, and clustering exercises. This article explores findings related to the discourse of ‘normality’ and is thus focused on a subset of the data from the study.

Results

Fifty-one PwH, aged 1.5 to 82 years, were interviewed and followed in their daily lives. Six treatment centres were visited, and 18 HCPs were interviewed. The study found that a discourse of present day ‘normality’, as compared to a difficult past, is ingrained in the haemophilia community. As a result, unlike most older PwH (40+), younger PwH (under 18) are not always taught to acknowledge the severity of their condition or how to sense bleeds (disease-related embodied knowledge), and risk unknowingly doing long-term damage to their bodies. Twenty-seven per cent (n=7/26) of younger PwH (children, teenagers) in the study were observed or described as engaging in high-risk behaviours in the short term indicating a lack understanding of long-term consequences.

Conclusions

These findings suggest that the discourse of ‘normality’ presents a number of challenges that need to be addressed, namely the potential for younger PwH to be unaware of bleeds and the general underreporting of haemophilia-related complications and limitations. One way forward in realising the full potential of advanced treatment could be to teach young PwH, through evidence-based initiatives, how to develop an embodied sense of their bleeds. Furthermore, if the current state of life with haemophilia is accepted as finally ‘normal’, then progress in further improving care may be stalled. It is important that remaining and new challenges are recognised in order for them to be acted upon.

Abstract

Introduction

People with haemophilia (PwH) experience recurrent bleeds in weight-bearing joints. Optimal management for people with severe haemophilia involves prophylaxis with factor VIII or IX, which helps to reduce the risk of bleeds and joint damage. However, this is expensive and frequently not an option in economically developing countries, where on-demand treatment is more commonly used as bleeding occurs. PwH with moderate and mild haemophilia are also treated on demand. Pain from bleeds and arthropathy is common in PwH; it is recognised as a burden that impairs quality of life and can be challenging to manage.

Aims

This study aims to establish greater understanding of the experience of pain in PwH in different countries, the factors that influence this, and how pain is currently managed.

Methods

PwH attending haemophilia treatment centres (HTCs) completed an anonymous questionnaire about their experience of pain and pain-relief within the previous 28 days (up to 10 PwH per participating HTC).

Results

209 PwH from 20 HTCs in 11 countries participated in the study. The median age was 36 (range 8–84); 181 (86.6%) had haemophilia A, 25 (12.0%) haemophilia B, and three (1.4%) did not know; 148 (70.8%) had severe haemophilia, 28 (13.4%) moderate, and 31 (14.8%) mild. Twenty-eight (13.4%) had an inhibitor. The majority (n=121; 57.9%) were on prophylaxis; 61 (29.2%) were treated on demand; 20 (9.6%) used a combination; 7 (3.3%) did not know. 154 PwH (73.9%) experienced a total of 1,945 days of pain with severity on a visual analogue scale reported as 4.5. The most commonly reported sites of pain were joints and muscles. There was no significant difference in pain incidence between countries. Children aged less than 16 years reported the lowest amount of pain, with reported pain increasing with age in older respondents. Simple analgesia such as paracetamol was used but participants reported that it did not relieve pain. Alternative pain-relief strategies including rest, physiotherapy, walking aids, alcohol or marijuana were also used with varying effect.

Conclusions

Pain is common among PwH and increases with age. Age and developmentally appropriate pain assessment should be a part of routine haemophilia care.

Abstract

Background

With the major advances in treatment of haemophilia in recent decades, people with haemophilia (PwH) are more protected in their daily lives than ever before. However, recent studies point to persisting or increasing patient experience of uncertainty.

Aims

The aim of this article is to further investigate findings related to how PwH understand and cope with uncertainty around their protection in their everyday life, one of the main themes identified in a large-scale ethnographic study of the everyday life of PwH, including beliefs and experiences related to their condition, their treatment, and their personal ways of managing the condition.

Methods

The study used ethnographic research methods. Five haemophilia experts provided historical and disease area context prior to the initiation of field research. During field research, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of the interactions of PwH with friends, family, and health care professionals (HCPs). Study researchers also conducted on-site observation at haemophilia treatment centres (HTCs) and interviewed HCPs. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenge mapping, and clustering exercises. This article explores findings related to uncertainty and thus focuses on a subset of the data from the study.

Results

Fifty-one PwH in Italy, Germany, Spain, UK, and Ireland were interviewed and followed in their daily lives, and 18 HCPs were interviewed. Fifty-two per cent (n=26/50) of PwH in the study experience difficulties translating clinical understanding of protection into specific activities in everyday life. Many have developed their own mental models and care adaptations to navigate treatment uncertainy: these seldom match the medical community's view. These mental models of protection among PwH can cause distress and influence behaviour in a way that can limit possibilities, and/or increase risk. There is also a prevalent tension in the strategies PwH have for managing their protection in terms of day-to-day vs. long-term ambitions.

Conclusions

These findings on PwH's experience of treatment uncertainty suggest a need to develop tools and communication materials to help PwH better understand the protection provided by their treatment regimen and what that means practically for everyday life.

Abstract

Background

Recent improvements in approaches to treatment have opened a window of opportunity to redefine and expand the goals of treatment in haemophilia This article explores treatment culture in light of these improvements and its potential impact on the range of possibilitis in the lived experience of haemophilia.

Aims

The aim of this article is to further investigate findings related to how health care professionals (HCPs) approach haemophilia treatment and care, one of the main themes identified in an ethnographic study of the everyday life of people with haemophilia (PwH). This large-scale study investigated PwH's beliefs and experiences related to their condition, their treatment, and their personal ways of managing the condition.

Methods

The study used ethnographic research methods. Five haemophilia experts helped frame the research design by providing historical and disease area context prior to the initation of field research. In the field, study researchers collected data through 8–12 hours of participant observation, semi-structured interviews, written exercises, facilitated group dialogues, and on-site observations of the interactions of PwH with friends, family, and HCPs. Study researchers also conducted on-site observation at haemophilia treatment centres (HTCs) and interviewed HCPs. The study employed a multi-tiered grounded theory approach and combined data were analysed using techniques such as inductive and deductive analysis, cross-case analysis, challenge mapping, and clustering exercises. This article explores findings related specifically to how HCPs approach haemophilia treatment and care, and is thus focused on a subset of the data from the study.

Results

Fifty-one PwH in Italy, Germany, Spain, UK, and Ireland were interviewed and followed in their daily lives. Eighteen HCPs from seven HTCs were interviewed, and on-site observation was undertaken at six of the HTCs. Most haematologists in the study ‘treated for stability’, rather than to guide PwH to overcome limitations. ‘Treating for stability’ here refers to an approach to haemophilia care that focuses on measuring success in terms of annual bleed rate, instilling a focus on mitigating risk, rather than an approach that allows PwH to overcome the limitations they face due to their condition. However, some haematologists had moved beyond treating for stability to instead treat for possibilities, enabling a better quality of life for PwH.

Conclusions

These results suggest that a culture of ‘treating for stability’ could be limiting progress in expanding the goals of treatment in haemophilia. Expanded metrics of success, more flexible approaches to treatment, and higher ambitions on behalf of PwH may be needed in treatment and care, in order for PwH to fully benefit from treatment advances and to increase their quality of life.

Abstract

Introduction

Switching between clotting factor products is becoming increasingly common as product choice increases and financial pressure grows to choose the most cost-effective options. Guidance on carrying out the switch recommends a complex and long process that may benefit from being defined in a protocol. Haemophilia nurses may be responsible for managing product switches; anecdotal evidence suggests that clinical practice is variable.

Aim

To explore the role of specialist nurses in switching between clotting factor products and their use of a protocol.

Method

Nurses attending the 2018 World Federation of Hemophilia Congress were surveyed about clinical practice at their treatment centre and use of a protocol for switching clotting factor products.

Results

Of 192 nurses attending the conference, 49 nurses returned completed questionnaires, 45 of which were included in the study after exclusions. Responses were exclusively from economically developed countries. Almost all respondents (96%) had direct experience of switching. Half of those who responded to a question about protocol-based switching reported that switches were based on a protocol. When authorship was reported, the protocol was written by haemophilia nurses in about half of cases. Practice about blood testing to determine individual pharmacokinetic parameters prior to the switch was variable, but most nurses (86%) reported screening for inhibitors prior to switching. Respondents agreed to share their protocols among their peers, although only four were received by the research team.

Conclusions

Clinical practice in switching between clotting factor products is variable. Some nurses are switching treatments for patients without the supported of a written protocol, whereas others are involved in writing and implementing protocols. Sharing protocols is a first step in helping to establish best practice.

Abstract

Background

Patients with haemophilia who are not adequately treated experience a lifetime burden of joint complications and loss of functional ability due to repeated bleeding episodes caused by low levels of clotting factor VIII or IX in the blood. These complications can significantly impact day-to-day life, including active participation in school and academic study in children with haemophilia (CwH). Treatment with factor replacement therapy can help to prevent this, but access to factor has been challenging in low-resource settings such as Northeast India. This study shows the impact of factor replacement therapy on bleeding episodes, joint complications and school absence among CwH in this setting.

Methods

A retrospective observational study was undertaken to examine the impact of receiving regular factor replacement therapy (prophylaxis or on demand) on school absences among CwH registered with the haemophilia treatment centre at Assam Medical College and Hospital. Annual bleed rate (ABR), Haemophilia Joint Health Score (HJHS) and Functional Independence Score in Haemophilia (FISH) were also assessed.

Results

Thirty-eight CwH were eligible for the study; 26 (68.4%) were on prophylaxis therapy and 12 (31.5%) received on-demand therapy. In the year before starting regular treatment, the mean ABR was 37.8 (+20.0), HJHS was 31.1 (+18.1) and mean FISH score was 21.1 (+4.2). At the end of the study period the mean (+SD) ABR in prophylactic therapy was significantly lower at 5.8 (+4.6) (p<0.001) and the HJHS was significantly lower at 4.7 (+4.6). FISH score significantly improved to 27.9 (+3.3) (p<0.001). Prophylaxis showed better (but not significant) results in comparison to on-demand therapy.

Conclusions

Treatment with factor replacement significantly reduces school absence in CwH and correlates strongly with joint health and functional improvement, with the effect slightly better with prophylaxis than on-demand therapy. Low dose prophylaxis is a good treatment option in low-resource settings, but improvements are also needed in rates of diagnosis.