Beyond the platelet count. The lifelong burden of immune thrombocytopenia
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Immune thrombocytopenia (ITP) is widely understood as a bleeding disorder defined by low platelets.
For the people who live with it, that definition is incomplete. Fatigue, cognitive symptoms, anxiety and the slow erosion of working and family life are often the heavier burdens, and none of them can be read from a blood test. Barbara Lovrencic, president of the Italian ITP patient association AIPIT, explains why care that looks past the count is long overdue
Written by Nicola Miller, RARE Revolution
Interview with Barbara Lovrencic, president, AIPIT (Associazione Italiana Porpora Idiopatica Trombocitopenica APS) and vice-chair, International ITP Alliance
Additional insights from: Mervyn Morgan, CEO, ITP Support Association
Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which the immune system destroys platelets, the cells the body needs to form clots. Diagnosis is one of exclusion, where no single test confirms the disease, and the same low platelet count produces varied outcomes and presentations in different people. One person may feel ‘well’ at platelet counts that would warrant urgent hospital admission and/or excessive bleeding in someone else.
That variability is why ITP needs to be treated as a condition of the whole person rather than a number on a lab report. The disease can occur at any age, but chronic ITP is more common in adults, and the evidence base for quality-of-life burden, including the international ITP World Impact Survey (I-WISh) of 1,507 adult patients and 472 physicians across 13 countries, sits almost entirely in the adult population1. Uncertainty begins at diagnosis. Twenty-two percent of I-WISh patients reported a delayed diagnosis, most often because of waits for additional tests or for specialist referral. Of those, over two-thirds felt anxious about the wait, and more than a third described feeling extremely anxious. Physicians surveyed estimated that up to a quarter of ITP patients are initially misdiagnosed, most often with drug-induced thrombocytopenia, aplastic anaemia or myelodysplastic syndromes1
Petechiae: pinpoint reddish-purple spots that look like a rash but are tiny bleeds under the skin.
Purpura: easy or excessive bruising without an obvious injury.
Frequent bleeding: nosebleeds, gum bleeds, or blood in the urine or stool.
Heavy menstruation: unusually heavy or prolonged menstrual flow.
(Source: Mayo Clinic)
The invisible burden patients carry every day
Bleeding and bruising are the visible signs of ITP. However, for many patients, there are deeper invisible symptoms too. During a four-month relapse in 2018, when her platelet count stalled at 4,000, Barbara Lovrencic, president, AIPIT (Associazione Italiana Porpora Idiopatica Trombocitopenica APS), explains how she found herself unable to support her son, who has type 1 diabetes. “I would be on our couch, and it was like climbing a hill just to move to the table and eat.” When operating his insulin pump, she recalls, “I couldn’t remember three-digit numbers. I had to go one digit at a time to avoid making an error.” These cognitive symptoms can be as disabling as physical ones, as she expands, “When I have very low platelets, I can’t remember numbers, and it becomes very difficult and exhausting to speak Italian, my second language.”
The I-WISh survey shows how widespread these symptoms are. Fatigue affected over half of adult patients at diagnosis, which was still present at the median of five years later. Of those who reported fatigue at diagnosis, two-thirds rated it severe, and when asked which symptom they most wanted resolved, almost half chose fatigue. Physicians, however, reported hearing about fatigue from only a third of their patients1.
The gap between what patients live with and what reaches the consultation room was one of the clearest findings of the study.
Anxiety follows the same pattern. Two-thirds of patients who reported anxiety about an unstable count rated it severe at diagnosis, falling very little five years on. Many feared their condition would worsen, and reported worries about dying—a figure that did not track with disease severity1. Barbara cites depression among people with ITP, and notes that it also does not automatically improve when the platelet count rises.
The disease also reshapes social and intimate life. In I-WISh, 70 percent of patients reported that ITP affected their social life and 60 percent that it affected their sex life.
One third expressed substantial concern about their physical appearance, reporting wearing long-sleeved clothing to hide bruising, even in warm weather1.
Importantly, a majority of physicians surveyed in I-WISh agreed that ITP symptoms reduce quality of life, but only 66 per cent agreed that ITP-related fatigue does so substantially.
How chronic disease reshapes a working life
Chronic ITP reshapes professional life in ways that rarely make it into the consultation. In I-WISh, nearly half of patients had reduced or seriously considered reducing their working hours because of the disease. A third had thought about leaving their job, with others reporting having declined a promotion or being forced to stop work altogether. Among patients still in employment at the time of the survey, many felt that ITP affected their workplace productivity.
Barbara recognises these patterns from the community she leads. “We have people who actually decided to retire early or switch from a full-time job to part-time, or even quit their job, especially if you have a job that’s demanding for travelling.” Treatment logistics add another layer. Some therapies carry food restrictions, others require injections, and patients who travel for extended periods can struggle to maintain a continuous supply.
Then there is ageing, a dimension that current research has barely begun to map. “Menopause is not addressed at all. I wouldn’t call us women in the menopause age, old, but we have this changing period that can be very, very challenging.” Comorbidities and drug interactions add new variables, and patients increasingly move between specialities as they grow older. “People with ITP who have other diseases very often are taken care of more by internal medicine doctors rather than haematologists. The educational part on ITP should be brought into other areas, not just haematology.”
The UK’s ITP Support Association points to the same gap in primary and generalist care, citing inadequate education among GPs and trainee doctors as one of the recognised unmet needs in the condition.
A childhood interrupted
For the proportion of children whose ITP persists, the early years can be defining. Diagnosed at aged four in Croatia, Barbara grew up in and out of hospital, with almost 300 hours of school absence in her third year of primary school alone. At diagnosis, her family was told to prepare for her funeral.
She describes a childhood marked less by bleeding than by exclusion. “All the kids of my neighbourhood came to understand that they couldn’t rough play with me and that I had to be away from the ball. I never did sports in school. Sometimes I felt like a glass doll, fragile and always in danger.”
Adolescence brings a different set of pressures. The instruction to be careful arrives precisely when young people are most invested in independence, identity, and belonging. Some withdraw; others reject the warning entirely. Barbara recalls a young man with ITP who, after a failed splenectomy and poor communication from his clinical team, stayed away from hospital for twenty years and took up high-risk activities, including riding a motorbike daily.
A further dimension, often overlooked, is the environment in which paediatric ITP is treated. Children are usually seen in oncology wards. “They see kids with cancer that they consider are much worse than them, so they feel like they don’t belong there, and like their problems are not ‘big enough’. They underestimate their own burden because they are compassionate towards other kids.” That habit of minimising can result in significant under-reporting of disease burden and carry into adulthood, shaping treatment decisions years later. Career consequences begin early. Some occupations are closed to people with ITP because of the bleeding risk, and the cumulative effect of school absences, sporting exclusion, and uncertainty narrows the horizon further. “It really asks from every patient to adapt and adjust their whole life plan.”
Why care must move past the platelet count
Physicians often recognise the wider impact of ITP, but treatment still hinges on platelets. Patient and physician priorities also diverge. When asked to rank treatment goals beyond a cure, patients in I-WISh placed healthy blood counts (64 percent), preventing episodes of worsening (44 percent), and increasing energy levels (41 percent) at the top. Physicians ranked reducing spontaneous bleeds first (72 per cent), followed by better quality of life and healthy counts. The clinical goal of preventing bleeding does not, on its own, capture what patients want from treatment1 .
The survey found that corticosteroids were routinely prescribed at diagnosis and again at first relapse. The harms of prolonged steroid exposure are well documented, and patient satisfaction with corticosteroids was the lowest of any treatment surveyed in I-WISh, at 53 percent. Satisfaction was substantially higher for other therapies, such as anti-CD20 therapies (79 percent), thrombopoietin receptor agonists (76 percent), and intravenous immunoglobulin (69 percent)1.
For Barbara, the problem is structural. “Sometimes we get overtreated, especially with steroids, which can do more harm than low platelets.” Effective ITP care, she argues, depends on three things in short supply across modern health systems. “It requires experience, it requires shared decision making and it requires time. Often, nowadays, physicians rarely have all three.”
The second structural gap is the absence of predictive biomarkers. Several treatment classes are now available, including thrombopoietin receptor agonists, anti-CD20 agents, SYK inhibitors, and more recent BTK inhibitors. The order in which they are tried is dictated by guideline-driven sequencing rather than by what is most likely to work for an individual patient.
“We still don’t know which patient will respond to which kind of therapy, so the trial-and-error approach continues. Some treatments, if started earlier, might have a greater probability of success, but by the time you arrive at the treatment that is right for you, you’ve lost time.”
The ITP Support Association identifies the same gap as a key unmet need: better tools to predict disease chronicity and treatment response, and longer-term clinical trial data measuring patient-reported outcomes rather than platelet counts alone.
Shared decisions, shared responsibility
Shared decision-making is endorsed in UK guidance and in international consensus, but progress in ITP is slow. In I-WISh, only 64 percent of patients felt their symptoms and quality of life were considered in treatment decisions, and just 74 percent felt their physician understood their treatment goals1. The remainder did not.
Closing that gap, Barbara argues, depends on a culture shift in which patient organisations are treated as partners. “We are witnessing a culture shift that is seeing patients and patient organisations partner with institutions, researchers, healthcare providers and industry. We are all part of one system, and we really need to work together.”
Better support is part of the picture. AIPIT runs a psychological support project for patients and caregivers, including monthly online meetings and individual therapy. The ITP Support Association reports that psychological support across the UK and Europe is constrained by funding and that digital health tools and educational resources for patients and clinicians remain uneven.
In an Italian quality-of-life project that included a narrative medicine component, participants were asked to describe living with ITP in one word. The most frequent answer was “storm”. For Barbara, the metaphor catches what a roller coaster cannot. “People used to say life with ITP is like being on a roller coaster, but I think it’s worse, because a roller coaster has a designed route and you can see what is in front of you. With ITP, we can’t.”
ITP cannot be defined by a platelet count alone. It is defined by the individual profile of symptoms, the family and working life around the patient, and the support, or lack of it, that surrounds the clinical pathway. As Barbara puts it: “What we all actually seek is to live the fullest possible life we can.” And until then, the work continues.
To learn more please visit:
AIPIT: www.aipit.com
ITP Support Association: www.itpsupport.org.uk
International ITP Alliance: www.globalitp.org
References
[1] https://pubmed.ncbi.nlm.nih.gov/33107998/
Articles are for information only and do not form the basis of medical advice. Individuals should always seek the guidance of their medical team before making changes to their treatment.
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