The complex immune dysregulation of ITP can impact platelet counts, bleeding, and HRQoL.1,3-6
In ITP, dysregulation of immune pathways results in increased platelet destruction and decreased platelet production.7

<h3>Bleeding occurs in around 60% of patients. Bleeding symptoms vary and can include8:</h3>

Petechiae and purpura, usually on the extremities1,9

black

Heavy menstrual bleeding1

Bleeding blisters and epistaxis1,9

Intracranial haemorrhage8

Learn more about how ITP can affect patients’ quality of life

Some ITP patients may experience systemic symptoms, including fatigue, fever, tachycardia, and cognitive effects, such as headaches and memory impairment.1,12-14

<h3>Severe bleeding occurs in 7% to 10% of adult patients and is typically correlated with lower platelet counts.10,11</h3>

<a href="${link:{uuid:{5951058e-a726-4620-a2dc-66df25bdf733},repository:{articles},path:{/Global/indications/itp/treating-itp}}}">Treatment of ITP</a> typically focuses on managing primary symptoms such as petechiae, purpura, and bleeding. However, patients with ITP often experience other symptoms such as fatigue and difficulties with memory and concentration that can profoundly affect their quality of life. That is why it is important to use treatment strategies that help improve HRQoL in ITP patients.1,12-14

<h3>Purpura and petechiae in ITP</h3>
ITP is an autoimmune blood disorder where easy bruising, petechiae and purpura often appear as common symptoms typically in lower extremities. Petechiae and purpura occur due to bleeding under the skin or mucous membranes.15,16
Petechiae in thrombocytopenia 
 Petechiae are small, non-blanching red or purple pinpoint lesions, usually less than 2 mm, that appear on the skin and mucous membranes. Petechial rashes result from bleeding into the skin’s dermal layer.15,17
Purpura in thrombocytopenia 
 Purpura refers to larger areas of cutaneous bleeding, which appear as purple or red patches on the skin that measure greater than 2 mm in size.15,17
In patients with ITP, purpura can appear due to even minor trauma or spontaneously due to low platelet levels. Purpura can also appear in mucosal membranes such as the hard palate, nasal septum, or gums, which can lead to bleeding gums.15,16,18

<h3>Other ITP bleeding symptoms and complications of thrombocytopenia</h3>
A low platelet count can lead to certain types of bleeding symptoms in ITP patients, including nose bleeds (epistaxis), gum bleeds (gingival), mouth blood blisters, blood in urine or stools, and heavy menstrual bleeding.9,19
Platelet levels &lt;10,000/μL could be associated with spontaneous widespread haematomas, and severe bleeding in ITP usually occurs when the platelet count is &lt;10 (x109/L). Severe bleeding can manifest as intracranial haemorrhages, gastrointestinal tract bleeds, and macroscopic haematuria. In some studies, severe bleeding occurred in approximately 7% to 10% of adult patients and typically correlated with lower platelet counts.10-12,16,20,21
ITP and intracranial haemorrhaging (brain bleed) 
 Intracranial haemorrhaging (ICH) is a rare and serious complication of ITP. The incidence of ICH in ITP patients is 1.54% in all ages.19,20,22,23
ICH symptoms that are a cause for concern include headache, persistent vomiting, altered mental status, and seizures. Risk factors for ICH include extremely low platelet counts of &lt;10,000/μL, head trauma, antiplatelet medication use, and severe bleeding.19,20,22,23

<h3>The symptoms of ITP go beyond bleeding</h3>
Learn more about the less visible symptoms of ITP and <a href="${link:{uuid:{9d762c77-664a-467f-a4f0-78211ab9453e},repository:{articles},path:{/Global/indications/itp/living-with-itp}}}">how they impact ITP patients’ quality of life</a>.&nbsp;

<h2>Primary ITP is a diagnosis of exclusion7,24,25</h2>
<ul>
 <li>Platelet counts &lt;100,000/μL can indicate ITP</li>
 <li>Platelet counts &lt;50,000/μL can indicate severe thrombocytopenia</li>
</ul>
<h3>But the lack of a specific test means other potential causes of thrombocytopenia must be ruled out7,24,26,27</h3>
Causes of secondary ITP include7,24,26,27:
<ul>
 <li>Chronic lymphocytic leukaemia (CLL)</li>
 <li>Helicobacter pylori infection</li>
 <li>COVID-19 infection</li>
</ul>
<h3>ITP is further classified as either newly diagnosed, persistent, or chronic27:</h3>

Low platelet counts are one facet of ITP, but when we look at it from another angle, we see the multiple dimensions of ITP. Immune thrombocytopenia (ITP) is a condition of complex immune dysregulation that reduces platelet counts—causing bleeding, bruising, fatigue, and other health-related quality-of-life (HRQoL) issues—while increasing inflammation and the risk of thromboembolic events (TEs).1-5

Defining ITP

IMMUNE-THROMBOCYTOPENIC-ITP

In a study, patients living with ITP rated their health-related quality of life (HRQoL) significantly worse than the general US population. They also reported worse HRQoL than patients living with hypertension, arthritis, or cancer, and similar to that of patients living with diabetes.1*
*The study used the Short-Form 36 questionnaire to evaluate the HRQoL of 73 adult ITP patients compared with that of the general US population and of patients with 6 other relatively common chronic disorders.1
<h3>ITP can impact patient HRQoL in multiple ways, including:&nbsp;</h3>

<h3>Fatigue</h3>
Reported as severe by patients with ITP, often regardless of platelet counts2

<h3>Heavy menstrual bleeding</h3>
Rated by patients and doctors as one of the most severe ITP symptoms2

<h3>Thromboembolic events (TEs)</h3>
Compared to patients without ITP, there is up to a 2x higher risk of TEs3

<h3>Cerebral microbleeds (CMBs)</h3>
Associated with lower platelet count and longer disease duration4

<h2>Fatigue is common and can be severe in patients with ITP2</h2>
In the Immune Thrombocytopenia World Impact Survey (I-WISh)* of 1507 patients and 472 physicians, patients rated functional, psychological, and emotional aspects of their condition. In I-WISh2:

58% of patients reported fatigue at diagnosis (n=870/1507),2&nbsp;

and 73% of those patients rated fatigue as severe (n=637/870)2

<h4>Even in patients with low disease activity, prevalence of severe fatigue exists, and fatigue persists even after platelet counts have increased. Disease activity alone does not explain variance in ITP.2,5</h4>
<h3>Factors that may play a role in fatigue5,6:</h3>
<ul>
 <li>Inflammation</li>
 <li>Physical and functional well-being</li>
 <li>Physical activity</li>
</ul>
Research shows that ITP is also linked to reduced activity and a notable increase in general, mental, and physical fatigue.7,8&nbsp;
The exact causes of fatigue and the underlying pathophysiology are not fully understood. However, ITP-related excessive and prolonged bleeding leads to iron deficiency and anaemia, which can be contributors to fatigue. Learn more about the <a href="${link:{uuid:{c74a8bbe-8c42-444b-b7ca-84850a1d0137},repository:{articles},path:{/Global/indications/itp/mechanism-of-disease}}}">causes and pathophysiology of ITP</a>.6,9,10
Furthermore, ITP-related activity restrictions can result in frustration, social withdrawal, and mood decline.7,11

—Male living with ITP, Germany

*I-WISh was an online, cross-sectional survey of patients with ITP and haematologists or haemato-oncologists who treat patients with ITP in 13 countries. The patient questionnaire collected information on demographics and diagnosis, symptoms of ITP, HRQoL and emotion associated with ITP, impact of ITP on work, finances, and support, treatment received, and patient and physician relationship. The physician survey comprised 6 sections that collected information on demographics, diagnosis of ITP and patient caseload, symptoms of ITP, impact of ITP on aspects of patients’ physical, emotional, HRQoL, social health, treatment patterns, and patient and physician relationship. There were 2 questions about the emotional impact of ITP, and the responses were based on a 7-point Likert scale (1 = never, 7 = a great deal). There was 1 additional question on the need for professional support.7

<h2>Heavy menstrual bleeding can have a major negative impact on patient HRQoL2</h2>

45% of women with ITP reported heavy menstrual bleeding at diagnosis2

and 83% of those said it was one of their most severe symptoms2

<h2>Thromboembolic events (TEs) are also more common among patients with ITP3</h2>

Patients with ITP face up to a 2X higher risk of TEs compared to patients without ITP3*
Risk factors for TEs in ITP12:
<ul>
 <li>Mechanisms of ITP itself</li>
 <li>Patient comorbidities and past history</li>
 <li>Current ITP treatments patient may be taking</li>
</ul>
*Adjusted risk ratio of 1.7 in a retrospective cohort study of 1140 patients with ITP and 5657 patients without ITP.3

<h2>Patients with ITP might also suffer from cerebral microbleeds4</h2>

CMBs were associated with lower platelet count and longer disease duration. The strong association between CMBs and ITP duration could indicate that CMBs may accumulate over time.4

In a 2020 study published in Blood,* 43% of patients with ITP had CMBs (n=21/49)4

*A cross-sectional prospective study of 49 patients with ITP and nadir platelet counts &lt;30,000/μL and 18 age-matched healthy controls that used susceptibility-weighted magnetic resonance imaging (MRI) to detect CMBs as a marker of occult haemorrhage.4

<h3>Neurological symptoms and cognitive effects of immune thrombocytopenia</h3>

Neurological manifestations in ITP without clinically significant bleeding are rare.14
However, patients with ITP might also suffer from cognitive impairment.14

50% of patients with ITP experience some level of cognitive impairment (N=69)13

Can ITP cause headaches?
Individuals&nbsp;with ITP can suffer from headaches and migraines. A sudden headache in ITP patients can also be an indication of the rare but severe complication of intracranial haemorrhage (ICH), which can cause other neurological symptoms.2,15-17

Patients reported significant quality of life (QoL) challenges related to their ITP1

Living with ITP

<h3>ITP Patient-Reported Outcomes Assessment Questionnaire (ITP-PAQ)5,6</h3>
ITP-PAQ is a validated tool that was designed to measure the HRQoL and treatment impact specifically for patients diagnosed with ITP.
The questionnaire typically evaluates various aspects of life affected by ITP, such as emotional well-being, and physical and social functioning. Additionally, the survey aids in assessing symptoms and their severity, including bruising and bleeding.&nbsp;
The ITP-PAQ facilitates a better overall understanding of how ITP affects individuals beyond clinical measures alone and their HRQoL.
<h3>ITP Life Quality Index (ILQI)1,6</h3>
The ILQI is a validated 10-item questionnaire that was developed to aid clinical discussions between patients and physicians about patients’ disease experiences, facilitating a patient-centric approach toward treatment decision-making. The ILQI typically involves surveys or questionnaires that patients complete, detailing their experiences, symptoms, and how these affect their quality of life.

<h3>Modified Fatigue Impact Scale (MFIS)2,7</h3>
The MFIS measures the impacts of fatigue on physical, cognitive, and psychosocial functioning.&nbsp;
This validated scale consists of a series of statements related to fatigue and its impact over a longer time, usually 4 weeks. The respondents rate their experiences over this period. The total scoring can then help healthcare providers understand the severity of fatigue and its effects on daily functioning. &nbsp;
In contrast to the MFIS, the daily fatigue impact scale (D-FIS) is a scoring tool to measure the short-term impact of fatigue in everyday life.
<h3>The Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-F)4,8</h3>
The FACIT-F is a validated scale that provides a 13-item tool to determine self-reported fatigue and its impact on daily activities and function.&nbsp;
The questionnaire includes questions regarding the intensity and frequency of fatigue, its effects on social and personal activities, and the overall energy level.&nbsp;
<h3>The Daytime Sleepiness Scale3</h3>
The daytime sleepiness scale is often represented by the validated Epworth Sleepiness Scale (ESS), a scale used to evaluate patients for excessive daytime sleepiness. The questionnaire measures how likely someone falls asleep in various situations.

For ITP patients, several scales are available to measure and track fatigue and its impact on quality of life. These tools can help patients and healthcare providers gauge symptoms and adjust their ITP treatment accordingly.1-4

Assessing fatigue in ITP patients

In ITP, immune dysregulation lowers platelet counts through multiple pathways. Autoreactive B cells produce autoantibodies that target platelets for destruction by macrophages and impair megakaryocyte maturation, reducing platelet production. Autoantibodies may also destroy platelets through other mechanisms.
Immune system activity can also lead to elevated levels of inflammatory markers.1-6

Adapted from Kashiwagi H, et al. Int J Hematol. 2013;98(1):24-33. © Japanese Society of Hematology (JSH).1

Treatments designed to achieve multi-immune modulation aim to better address the multiple dimension of ITP7,8

<h3>Watch the mechanism of disease (MOD) video to learn more about the multiple dimensions of ITP</h3>

Complex immune dysregulation can lead to autoimmunity and inflammation1-6

Mechanism of Disease

<h2>Bruton’s tyrosine kinase (BTK) plays a pivotal role in complex immune dysregulation9,10</h2>
B cells are responsible for the production of autoantibodies; macrophages are essential for phagocytosis; and the NLRP3 inflammasome plays a key role in systemic inflammation. All of these processes are regulated by BTK.9,10
<ul>
 <li>In B cells: The BTK pathway is crucial for proliferation, differentiation, and autoantibody production</li>
 <li>In macrophages: Various signalling pathways dependent on BTK drive phagocytosis, degranulation, and the production of inflammatory cytokines</li>
 <li>Systemically: BTK drives the production of mediators of inflammation like NLRP3 inflammasome, which can contribute to platelet destruction and systemic inflammation</li>
</ul>

Patients living with ITP have elevated inflammatory markers, including10:
<ul>
 <li>TNF-α and IFN-γ</li>
 <li>IL-2 and IL-6</li>
 <li>NLRP3 inflammasome cytokines IL-1β and IL-18</li>
</ul>
BTK is implicated in the production or activation of these inflammatory markers and is a critical driver of inflammation.10
<h3>Other signalling pathways involved in the pathogenesis of ITP8</h3>
With incremental understanding of molecular mechanisms driving ITP pathology, it is becoming increasingly clear that multiple signalling pathways are involved in the pathogenesis and manifestation of the disease.8

ITP is about more than platelets

BCR, B-cell receptor; Fc, fragment crystallisable; IFN, interferon; IL, interleukin; NLRP3, nucleotide-binding domain, leucine-rich repeat and pyrin domain-containing protein 3; TNF, tumour necrosis factor.

<h2>Causes and pathophysiology of ITP1-4,8,11,12</h2>
ITP has an estimated prevalence of up to 9.5 per 100,000 people. The causes and pathophysiology of immune thrombocytopenia (ITP), historically called idiopathic thrombocytopenic purpura or immune thrombocytopenic purpura, are multifactorial and trigger a wide range of symptoms.
The pathophysiology of ITP is complex, involving various immune cells and different organs. Platelet destruction and inhibition of thrombopoiesis (platelet production) are key drivers of the disease. Current treatments for ITP aim to target these mechanisms of immune thrombocytopenia.
Our understanding of the pathophysiology of ITP is expanding and opening up new treatment possibilities for more targeted therapies and overall disease control.
<h3>What are the causes of ITP12-14</h3>
The cause of ITP in adults can be differentiated by whether the disease is primary or secondary ITP.&nbsp;
In primary ITP, the underlying causes remain unidentified—this is why it was previously known as idiopathic thrombocytopenic purpura. This is the most common cause of ITP, with approximately 80% of ITP patients diagnosed with primary ITP.&nbsp;
In contrast, secondary ITP is caused by known factors such as certain medications, infections, or cancers.&nbsp;
It is often associated with persistent autoimmunity and underlying autoimmune diseases including systemic lupus erythematosus (SLE) and antiphospholipid syndrome (APS).&nbsp;
Chronic ITP is characterised by the persistence of symptoms for more than 12 months after the initial diagnosis. Chronic ITP is more common in adults than in children. Recent studies suggest genetic factors, including polymorphisms in cytokine receptor genes and genes regulating T cell activity, may influence the susceptibility for chronic ITP. However, more research is needed to fully understand the causes of chronic ITP.

Complex immune dysregulation can lead to autoimmunity and inflammation1-6

<h2>ITP treatment and management1,2</h2>
Many ITP patients without severe bleeding could be managed by observation and may only require pharmacological interventions when their platelet count falls below 20-30 (x109/L).1,2
More information on how healthcare providers can create an individualised ITP treatment plan can be found in the <a href="https://pro.campus.sanofi/global/itp/articles/treatment-guidelines">ITP diagnosis and treatment guidelines</a>.

<h3>Treating ITP with pharmacological interventions3-10</h3>
Current ITP therapies aim to address thrombocytopenia only, by preventing bleeding and increasing low platelet counts. Whilst ITP-related bleeding is common, only in rare cases is it life-threatening.&nbsp;
There remains an unmet need for therapies that address multiple targets contributing to <a href="${link:{uuid:{c74a8bbe-8c42-444b-b7ca-84850a1d0137},repository:{articles},path:{/Global/indications/itp/mechanism-of-disease}}}">the underlying immune dysregulation driving ITP</a> instead of just the primary symptoms.

<h4>Corticosteroids for ITP1,2,11</h4>
For many patients, corticosteroids are the recommended first-line treatment for ITP. The therapy may be initiated in newly diagnosed adult patients without severe bleeding when their platelet count falls below a threshold between 30 (x109/L) to 20 (x109/L).
Corticosteroids may ameliorate platelet counts by broadly suppressing the activity of the autoreactive immune system, thereby reducing the generation of autoantibodies against platelets.&nbsp;
The medication starts to exert its function within 2-14 days and has a response rate of 60-80%. However, only 30-50% of patients show a durable response when the medication is discontinued, and permanent remissions are uncommon.&nbsp;
Corticosteroids may cause side effects in most patients, including negative impact on mental health, hypertension, muscular atrophy, and glaucoma.
<h4>Thrombopoietin receptor agonists (TPO-RA) for ITP</h4>
TPO-RA are usually indicated for second-line treatment in treatment-resistant patients with new, persistent, and chronic ITP. They can also be used as an emergency treatment.1,2,11,12&nbsp;
TPO-RA stimulate thrombocytopoiesis in megakaryocytes. Effects are seen after 3-14 days depending on the specific drug used, with response rates varying between 65-80% and only 8-32% of patients experiencing a durable response after stopping the medication.2,11,12
TPO-RA cause various side effects such as thrombosis, transaminitis skin reactions, and gastrointestinal problems.2,11,12

<h4>Targeted immunotherapy for ITP1,2,11,12</h4>
Targeted immunotherapy is used as a second- or third-line treatment. These drugs might be a suitable option for patients who want to avoid long-term medication and surgery.&nbsp;
Targeted immunotherapy uses antibodies to target the autoantibody-producing B cells leading to the destruction of the cells.&nbsp;
This therapy starts to exert its function after 7-56 days. 60% of patients initially respond to the treatment, with the effect sustained in 10-40% after the drug is discontinued.&nbsp;
Side effects are usually mild; however, treatment could make patients more susceptible to infections.&nbsp;
<h4>Intravenous immunoglobulin (IVIg) for ITP1,2,13,14</h4>
IVIg quickly elevates platelet levels and could therefore be used as an emergency treatment for ITP. As a first-line treatment, IVIg could also be combined with corticosteroids. However, IVIg treatment for ITP alone has not been reported to provide a durable response.&nbsp;
The detailed mechanism of action of IVIg is currently unknown. They are thought to inhibit phagocytosis of platelets.&nbsp;
Clinically relevant side effects of IVIg can include low blood pressure, arrhythmia, skin reactions, and flu-like symptoms.
<h4>Anti-D immunoglobulin (anti-D) for ITP1,2</h4>
Anti-D immunoglobulin is only available in some countries for the treatment of ITP. It could be used as a first-line treatment. However, the potential side effects could render it unsuitable for most patients without severe bleeding.&nbsp;
Anti-D is an antibody that attaches selectively to rhesus-positive erythrocytes. The interaction leads to the degradation of the erythrocytes by the spleen. The process is thought to interfere with the destruction of platelets and consequently cause an increased platelet count in patients with immune thrombocytopenia.&nbsp;
Serious side effects of anti-D treatment for ITP include intravascular haemolysis.&nbsp;

<h3>Non-pharmacological treatments of ITP</h3>
In addition to medications, non-drug-based interventions are available.

<h4>Splenectomy for ITP1-3,11,12,15</h4>
Splenectomy can be a useful second-line treatment for chronic patients who prefer to avoid long-term medication and aim for a durable response—the success rate of splenectomy for ITP is approximately 80%. A splenectomy should be avoided in the first 12 months of the initial diagnosis since ITP can go spontaneously into remission during the first year. Furthermore, ITP relapse or refractory ITP after a splenectomy affects 30-50% of patients.&nbsp;
A splenectomy can also be used as an emergency treatment.&nbsp;
The spleen plays a dual role in ITP. Splenic macrophages clear platelets covered in autoantibodies while splenic plasma cells are the main producers of autoantibodies against platelets. Therefore, removing the spleen interrupts these processes.
Side effects for ITP patients post-splenectomy include an increased risk of infection with encapsulated bacteria and require repeated vaccinations. A large registry-based study showed a higher risk of both early and late sepsis among patients with ITP who had undergone splenectomy than among those who did not. A higher incidence of other vascular complications, such as coronary artery disease, stroke, and chronic thromboembolic pulmonary hypertension, has also been reported among patients who have undergone splenectomy.
<h4>Platelet transfusion for ITP2,11,12,16,17</h4>
Platelet transfusion in combination with IVIg and corticosteroids can be used as an emergency treatment.&nbsp;
The effect of platelet transfusions wanes quickly. In addition, the treatment appears to fail in reducing mortality, and there are only limited studies on the benefits of platelet transfusions in ITP.&nbsp;
It’s important to note that platelet transfusions can induce severe immune responses such as anaphylactic and febrile non-haemolytic transfusion reactions.&nbsp;

<h4>Medication induced ITP18,19</h4>
Some drugs can trigger ITP. Medications that can induce ITP include quinine, penicillin, heparin, gold salts, sulfonamide antibiotics, and non-steroidal anti-inflammatory drugs. In these cases, stopping the medication should be considered for the management of ITP.
<h4>Lifestyle2</h4>
Some ITP patients may have to adopt lifestyle changes to prevent bleeding. This could include avoiding activities that are associated with an increased risk of bleeding, such as combat and contact sports.

<h3>Emerging treatments for ITP</h3>
Bleeding is only one consequence of ITP, but there is a wide range of <a href="${link:{uuid:{9d762c77-664a-467f-a4f0-78211ab9453e},repository:{articles},path:{/Global/indications/itp/living-with-itp}}}">ITP symptoms</a>&nbsp;that impact the health-related quality of life (HRQoL) of patients.3,20-23
Treatment goals in ITP have shifted from targeting only platelet counts to comprehensive disease control.2
An improved understanding of ITP disease pathophysiology is enabling researchers to develop innovative treatments that target the underlying mechanisms of ITP. Among these are Bruton’s tyrosine kinase (BTK) inhibitors, neonatal Fc receptor (FcRn) blockers, recombinant immunoglobulin multimers, complement inhibitors, plasma-cell–depleting therapies, and B cell activating factor (BAFF) inhibitors.7,11
Learn more about:

Immune thrombocytopenia, or immune thrombocytopenic purpura (ITP), treatment in adults needs to be individualised for each patient based on various factors such as the duration of the disease, age, general health, and importantly, patient preference.1,2

Treating ITP

<h3>ITP treatment guidelines for adults1-3</h3>
The ideal treatment for ITP varies between different age groups. Therefore, healthcare providers must consider the patient’s age when preparing a treatment plan. Below is a summary of ASH recommendations on how to treat ITP in adults.
<a href="https://ashpublications.org/bloodadvances/article/3/23/3829/429213/American-Society-of-Hematology-2019-guidelines-for" target="_blank">ASH recommendations</a> mainly cover primary immune thrombocytopenia guidelines, where the primary cause of ITP is unknown. In secondary ITP, the cause of the disease (eg, an infection or medication) is known and first-line treatment often addresses the pre-existing condition.&nbsp;

<h3>First-line ITP treatment1</h3>
Key decisions for healthcare providers treating ITP are criteria for admitting a patient to the hospital and when to start treatment.&nbsp;
Newly diagnosed patients with a platelet count below 20 (x109/L) without life-threatening bleeding should be admitted to the hospital, whilst patients with an established ITP diagnosis or a platelet count exceeding 20 (x109/L) can be treated at home.&nbsp;
Not all patients with ITP require medical interventions. Asymptomatic patients with non-life-threatening bleeding and a platelet count exceeding 30 (x109/L) can be managed with observation. In contrast, first-line treatment should be considered for all patients when their platelet count falls below 30 (x109/L).&nbsp;
Management of adult ITP patients with non-life-threatening bleeding1:
<figure class="table" style="width:100%;">
 <table class="ck-table-resized">
 <colgroup>
 <col style="width:17.51%;">
 <col style="width:41.16%;">
 <col style="width:41.33%;">
 </colgroup>
 <tbody>
 <tr>
 <td>&nbsp;</td>
 <td style="text-align:center;" colspan="2">Initial pharmacological intervention vs observation</td>
 </tr>
 <tr>
 <td>Clinical criteria</td>
 <td style="text-align:center;">Platelet count&nbsp; 
 &lt;30 (x109/L) 
 in 
 Newly diagnosed ITP patients 
 who are asymptomatic or have minor 
 mucocutaneous bleeding</td>
 <td style="text-align:center;">Platelet count&nbsp; 
 ≥30 (x109/L) 
 in 
 Newly diagnosed ITP patients 
 who are asymptomatic or have minor 
 mucocutaneous bleeding</td>
 </tr>
 <tr>
 <td>Management recommendation</td>
 <td style="text-align:center;">Start of first-line treatment</td>
 <td style="text-align:center;">Observation</td>
 </tr>
 <tr>
 <td>&nbsp;</td>
 <td style="text-align:center;" colspan="2">Hospital admission vs home management</td>
 </tr>
 <tr>
 <td>&nbsp;</td>
 <td style="text-align:center;">Platelet count&nbsp; 
 &lt;20 (x109/L) 
 in 
 Newly diagnosed ITP patients 
 who are asymptomatic or have minor 
 mucocutaneous bleeding</td>
 <td style="text-align:center;">Platelet count&nbsp; 
 ≥20 (x109/L) 
 in 
 Newly diagnosed ITP patients 
 who are asymptomatic or have minor 
 mucocutaneous bleeding</td>
 </tr>
 <tr>
 <td>&nbsp;</td>
 <td style="text-align:center;">Inpatient treatment</td>
 <td style="text-align:center;">Outpatient treatment</td>
 </tr>
 </tbody>
 </table>
</figure>

<a href="${link:{uuid:{5951058e-a726-4620-a2dc-66df25bdf733},repository:{articles},path:{/Global/indications/itp/treating-itp}}}">First-line treatments for ITP</a> include corticosteroids, intravenous immunoglobulin (IVIg), and anti-D immunoglobulin. Corticosteroids are deemed a suitable first-line treatment for most ITP patients. Importantly, all first-line treatment decisions should consider the patient’s preferences.1,2,4 &nbsp;
The impact of ITP on patients goes beyond platelet burden and can severely impact a person’s quality of life. The treatment therapies or treatment modalities of ITP can also adversely affect patients. For instance, corticosteroids can cause hypertension and negatively influence sleep and mental health.1,2,4
Treating healthcare providers should consider regularly assessing patients living with ITP and their health-related quality of life (HRQoL). Learn more about <a href="${link:{uuid:{c74a8bbe-8c42-444b-b7ca-84850a1d0137},repository:{articles},path:{/Global/indications/itp/mechanism-of-disease}}}">assessments of patients living with ITP</a>. 1,2,4&nbsp;

<h3>Advanced therapy use for ITP</h3>
Some patients do not respond to corticosteroids or experience ITP for more than 3 months.1
For these patients, a change in the treatment plan should be considered. <a href="${link:{uuid:{5951058e-a726-4620-a2dc-66df25bdf733},repository:{articles},path:{/Global/indications/itp/treating-itp}}}">Second-line ITP treatment</a>&nbsp;options include rituximab, splenectomy, and thrombopoietin receptor agonists (TPO-RA).1
The guidelines provide comprehensive considerations for selecting the best second-line treatments for ITP. Patients who are opposed to long-term medication may benefit from a splenectomy or treatment with rituximab. However, ITP patients can spontaneously go into remission within the first year,1 in which case a splenectomy should be avoided in this period. On the other hand, TPO-RAs might be a suitable option for patients who place a high value on achieving a durable response or avoiding surgery.1
In patients with additional risk factors, some treatments may elevate the risk of thrombosis and thromboembolism.5,6
Considerations influencing treatment discussions1:
<ul>
 <li>Adherence to medication</li>
 <li>Age of the patient</li>
 <li>Availability</li>
 <li>Comorbidities</li>
 <li>Cost</li>
 <li>Duration of the disease</li>
 <li>Frequency of bleeding</li>
 <li>Patient preferences</li>
</ul>
Overall, more treatment options are needed in ITP. Response rates and durability of current treatments can vary widely whilst some also have unfavourable side effect profiles or dosing regimens. Learn more about <a href="${link:{uuid:{8eff7d37-c8d4-477c-a6b5-52e04965a9b6},repository:{articles},path:{/Global/indications/itp/unmet-needs}}}">the future of ITP treatments</a>.7,8&nbsp;

<h3>ITP treatment algorithm</h3>
Making a second-line treatment plan for ITP is complex as patient preferences and different clinical factors must be considered.1,2&nbsp;
This treatment algorithm for adult patients can help provide general guidance on managing persistent or first-line treatment-resistant ITP. 1,2&nbsp;

Figure 1. Algorithm for the selection of second-line therapy in adults with ITP. Selection of second-line therapy in adults with ITP should be individualised based on duration of disease and patient values and preferences. Other factors that may influence treatment decisions include frequency of bleeding sufficient to require hospitalisation or rescue medication, comorbidities, compliance, medical and social support networks, cost, and availability of treatments. Patient education and shared decision-making is encouraged. Numbered recommendations corresponding to each treatment option are provided.1

<h3>Limitations of the ASH ITP treatment guidelines for adults</h3>
The guidelines only cover patients who have been already diagnosed with ITP and exclude guidance on treatments approved after 2017 and patients who are pregnant or haemorrhaging.1
<h3>Do other ITP guidelines agree?</h3>
Guidelines for ITP can differ slightly between countries. However, most guidelines are aligned on treatment approaches, including2,6:
<ul>
 <li>An emphasis on incorporating patient preference into treatment decisions and discussing the potential impact of different treatments on quality of life.</li>
 <li>Acknowledging that preferences of patients with chronic ITP may evolve during the course of the disease and recommending ongoing discussions as a routine part of follow-up.</li>
</ul>

ASH, American Society of Hematology; IVIg, intravenous immunoglobulin; HRQoL, health-related quality of life; ITP, immune thrombocytopenic purpura.

The American Society of Hematology (ASH) guidelines provide evidence-based guidance on the management of adult patients with newly diagnosed and chronic immune thrombocytopenia (ITP). This guidance was delivered by an ITP expert panel consisting of clinical specialists and patients.1

Treatment Guidelines

<h3>In a study, 75% of ITP patients felt their condition was not well-managed from a HRQoL perspective.3</h3>
Current treatment goals in ITP are to stop active bleeding and reduce the risk of further bleeding. Response rates for ITP treatments vary widely as measured by improved platelet counts, and the mechanisms of action (MOA) of current treatments may not fully address the complex nature of the disease.1
Additional rescue medication is often necessary for patients with ITP.4

—Female living with ITP, UK

<h2>Investigational therapies for ITP</h2>
Ongoing research aims to better address the multiple dimensions of ITP by targeting the underlying pathophysiology of the disease. Several clinical trials are in progress investigating novel drugs for treating ITP.5,6

Bruton’s tyrosine kinase (BTK) inhibitors, which inhibits macrophage phagocytosis leading to a decrease in platelet destruction, prevent the production of autoantibodies, and may inhibit inflammation5,7-9

Neonatal Fc receptor (FcRn) blockers, which increase platelet antibody clearance, leading to a decrease in peripheral platelet destruction and immune response5,10

Recombinant immunoglobulin multimers, which decrease platelet destruction and increase antiplatelet antibody clearance6

Complement inhibitors, which decrease complement-dependent cytotoxicity11

Plasma cell-depleting therapies, which decrease antiplatelet antibodies6,11

B cell activating factor (BAFF) inhibitors, which reduce B cell survival and consequently decrease antiplatelet antibodies12,13

<h3>Bruton’s tyrosine kinase (BTK) inhibitors5,7-9</h3>
BTK is an enzyme expressed mainly in B cells, myeloid cells, and platelets. BTK is crucial for the signalling pathways that regulate the development and maturation of B cells and the overall immune response.
Several BTK inhibitors targeting these mechanisms have been approved or are currently in development. BTK inhibitor (BTKi) therapy can prevent macrophage phagocytosis in ITP, leading to a decrease in platelet destruction; can prevent production of autoantibodies; and may also inhibit inflammation.
However, patients can develop a resistance to BTK inhibitors when mutations in the targeted cell occur. Newer generations of BTK inhibitors aim to tackle BTK inhibitor resistance and common class AEs with various strategies.&nbsp;
<h3>Neonatal Fc receptor (FcRn) blockers5,10</h3>
FcRn regulates the immune response by presenting immunoglobulin G (IgG) antigens to immune cells and regulating IgG levels in the plasma.
FcRn blockers in ITP may increase platelet antibody clearance, leading to a decrease in peripheral platelet destruction and immune response.&nbsp;
<h3>Recombinant immunoglobulin multimers6</h3>
Recombinant immunoglobulin multimers decrease platelet destruction and increase antiplatelet antibody clearance. Recombinant immunoglobulin multimers bind to Fc receptors on immune cells, preventing the attachment of pathogenic antibodies to these receptors. As a consequence, in conditions like ITP, the immune-mediated destruction of platelets could be reduced and platelet counts increase.

<h3>Complement inhibitors6,11</h3>
Complement inhibitors decrease complement-dependent cytotoxicity. Complement inhibitors block the activation of the complement system, a part of the immune response that marks cells, such as platelets, for destruction. By inhibiting specific complement pathways, these agents may potentially reduce platelet destruction in autoimmune conditions like ITP.&nbsp;
<h3>Plasma-cell–depleting therapies6,11</h3>
Plasma-cell–depleting therapies decrease antiplatelet antibodies. Plasma-cell–depleting therapies target and reduce plasma cells, helping to minimise platelet destruction and improve platelet counts. However, plasma-cell–depleting therapies may fail to completely eliminate long-lived plasma cells that regulate antiplatelet autoantibody production.&nbsp;
<h3>B cell activating factor (BAFF) inhibitors12,13</h3>
BAFF binds to its receptor on B cells promoting the survival of B cells and initiating the differentiation of the B cells to immunoglobulin-producing plasma cells. Both are crucial factors in ITP pathogenesis.
BAFF inhibitors may reduce B cell survival and consequently decrease antiplatelet antibodies. Thus, in conditions like ITP, BAFF inhibitors could help to preserve platelet counts.
TPO-RA, thrombopoietin receptor agonist.

More treatment options are needed for patients with ITP1,2

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