Living, Caring, Learning – Collaborating across borders to support a haemophilia patient with a rare complication

Moyo (a pseudonym) has moderate haemophilia A. As a young man, he had a serious accident at work and subsequently developed an iliopsoas muscle bleed. This was inadequately treated due to challenges in accessing coagulation factor concentrates (CFCs). At the time, the supply of CFCs we had access to in Kenya was solely through donations from the World Federation of Hemophilia (WFH) and Indiana Hemophilia and Thrombosis Centre (IHTC) ^[2,3].

Moyo went on to develop a large swelling in his lower abdomen and his health deteriorated. On further assessment we established he had a pseudotumour, a rare complication of haemophilia where an encapsulated haematoma forms following recurrent bleeding. It was the first time the team had seen this complication and thanks to our AMPATH partnership we were able to get expert advice from the IHTC team. We also had discussions with specialists in other HTCs including in Italy, Spain, Argentina and Israel. Everyone agreed that surgery as an option was too risky, and we started to treat Moyo with factor VIII (FVIII) prophylactically to try to shrink the pseudotumour ^[4].

As a country, we did not have enough CFCs to meet our on-demand treatment needs, let alone support a prophylaxis programme. The limited and intermittent supply meant we just couldn’t access enough FVIII to sustain prophylaxis for Moyo. This meant the outcomes were not as we had hoped and the pseudotumour continued to grow. Moyo’s physical activity was extremely limited due to the pain and swelling it caused. We were very concerned as it was also displacing and compressing adjacent internal organs, threatening to compromise their function. Home visits were an important part of the care we could give, and we saw that the situation put a lot of pressure on both Moyo and his family. In addition to his treatment needs and the debilitating physical impact of the pseudotumour, it was evident that he needed support for his psychological wellbeing and the social and financial impacts too.

At this time, the IHTC had initiated a clinical trial for treating pseudotumours with emicizumab. They very generously flew Moyo to the USA for further assessment and treatment, and he was enrolled in the trial. Emicizumab mimics FVIII but behaves slightly differently – it has a longer half-life and provides constant coagulant activity equivalent to FVIII levels of 15-20% ^[5]. It was hoped that prophylaxis with emicizumab would prevent further bleeding into the pseudotumour, allowing it to shrink over time, improving the pain and swelling Moyo was suffering, and relieving the compression on his other organs.

When Moyo returned home, he was followed up by our research team at MTRH, in line with the trial protocol. Sadly, again, things didn’t go as hoped. Moyo had frequent bleeds despite prophylaxis with emicizumab and the pseudotumour increased in size. On top of this, CFCs to treat breakthrough bleeding remained in very short supply. After 2.5 years, he was withdrawn from the trial.

Since this time, the supply of CFCs through the WFH Humanitarian Aid programme has increased and Moyo has been able to resume prophylaxis with FVIII infusions. The pseudotumour is stable and he has reported feeling better. He continues to be cared for by the team at MTRH. Progress is also now being made to ensure the Kenyan government is able to offer expensive coagulation factors to the people who need them.

My involvement in Moyo’s care taught me a lot about the importance of a holistic approach – not just considering his physical needs but also the wider impact on his wellbeing and that of his family. Home visits as part of outreach activities are an integral part of healthcare delivery. I learned a lot about being compassionate and gaining greater understanding of what our patients go through.

This situation also challenged me to look further afield for help for patients and not to be limited by the space in which we work. If we don’t know what to do for a patient there is always somebody somewhere who has the experience to advise the best course of action. If we hadn’t explored all the opportunities for Moyo, I don’t think he would have received the care he needed. Even though emicizumab didn’t work for him, he knew that we were all behind him, doing everything we could to help – not just us, but his family, friends and community too. I think all of this contributed to the fact that he feels better now and is getting on with his life. He needs crutches to get around but he now runs his own small business and he can support his family. He is building a freedom for himself, even though we couldn’t achieve the perfect outcome for him.

Informed consent has been obtained from the patient discussed in this paper.

The authors have advised no interests that might be perceived as posing a conflict or bias.

With thanks to Anne Greist of the Indiana Hemophilia and Thrombosis Centre.

Writing support was provided by Jenny Bryan.