Israeli team develops way to find genetic flaws in fetus at 11 weeks
What Is Thrombocytopenia?
Medically reviewed by Archana Sharma, DO
Thrombocytopenia is a platelet disorder that occurs when your blood platelet count drops too low. In adults, a normal platelet count typically ranges from 150,000 to 450,000 platelets per microliter of blood. However, if the count falls below 150,000, it is considered lower than normal.
Platelets, which are also called thrombocytes, are produced in the bone marrow from larger cells. When we get injured, these platelets team up and form a plug to seal the wound, called a blood clot or thrombus.
When your platelet count is low, it can become challenging to stop bleeding effectively. Bleeding can occur internally within the body, underneath the skin, or on the skin's surface. Serious bleeding complications usually arise when your platelet count becomes very low. Thrombocytopenia can potentially be life-threatening, especially if you have serious bleeding or are bleeding from the brain. It is crucial to seek early medical treatment to prevent serious complications.
Platelets can also activate when they sense a foreign invader in the body, releasing proteins to fight the infection. In addition to an increased likelihood of not being able to stop bleeding, thrombocytopenia may also lower your body's chances of successfully fighting the infection.
Researchers aren't exactly sure how many people have thrombocytopenia, as it is very likely underdiagnosed and underreported, and many people with it may be experiencing only mild symptoms.
Symptoms of ThrombocytopeniaThe main indicators of thrombocytopenia are associated with bleeding. You may not experience any symptoms, but if you do these symptoms can manifest suddenly or gradually develop over time. The condition is often discovered through a routine blood test.
Common signs of bleeding in thrombocytopenia may include:
Prolonged bleeding, even from minor injuries
Petechiae, which are small, flat red spots beneath your skin caused by leakage from your blood vessels
Purpura, or bleeding in the skin, resulting in red, purple, or brownish-yellow spots
Nosebleeds or bleeding from the gums
Blood in the urine (hematuria) or stool (hematochezia), causing it to appear as bright red or as a dark, tarry color
Heavy menstrual bleeding
Bruising
Thrombocytopenia is generally caused by one of three things. These are insufficient platelet production in your bone marrow, increased breakdown of platelets within your bloodstream, and increased breakdown of platelets in your spleen or liver.
Other conditions and factors can affect platelet production in you bone marrow, including:
Aplastic anemia, which occurs when your bone marrow fails to produce an adequate number of blood cells
Bone marrow cancers like leukemia
Cirrhosis, a condition characterized by liver scarring
Folate deficiency
Rare infections affecting the bone marrow
Myelodysplastic syndrome, which occurs when the bone marrow either makes too few healthy blood cells or produces defective cells
Vitamin B12 deficiency
Thrombocytopenia can also occur through the use of certain medications, including chemotherapy. A 2021 study reviewed data from over 15,000 patients and found that 13% of those being treated for solid tumors and 28% of those with haematologic malignancies, such as leukemia and Myelodysplastic syndrome, developed thrombocytopenia within the first three months of initiating chemotherapy.
Environmental exposure to toxic chemicals like pesticides, arsenic, and benzene can also impede healthy platelet production. Unhealthy lifestyle habits, such as excessive alcohol consumption, can temporarily lower your platelet count, especially in people with low vitamin B12 or folate levels.
DiagnosisTo diagnose thrombocytopenia, your healthcare provider will start by gathering information about your medical history and family background. They will also ask you about any symptoms you may be experiencing, and perform a physical examination to identify any signs of bleeding.
Your provider may think it's necessary to perform more blood tests. For example, a complete blood count, or CBC, may be ordered. This test measures the levels of platelets and other blood cells in your bloodstream. Another test your provider may order is a blood smear test. This involves placing a small sample of your blood on a slide and examining it under a microscope to further examine your platelets. Additionally, bone marrow tests may be ordered to assess the overall health of your bone marrow.
If your healthcare provider suspects that you have thrombocytopenia or another bleeding disorder, they may refer you to a hematologist. Hematologists specialize in diagnosing, treating, and managing blood-related conditions.
Treatments for ThrombocytopeniaThe treatment approach for thrombocytopenia varies depending on the underlying cause and the presence of symptoms. In cases of mild thrombocytopenia, treatment may not be necessary. However, if you are experiencing severe bleeding or have a high risk of complications, your healthcare provider may recommend various medications or procedures. Treating the underlying condition responsible for the low platelet count is also important, and will be part of the treatment regimen.
MedicationsSometimes medications can cause platelet levels to drop. If a reaction to a medication is the culprit behind your low platelet count, your provider may prescribe an alternate medication. For cases where your immune system is causing your low platelet count, your provider may prescribe medications that suppress your immune response (immunosuppressants).
Medications such as corticosteroids (e.G., prednisone) are commonly used to increase platelet count. Other medicines like Promacta (eltrombopag) and Nplate (romiplostim) can help stimulate platelet production. Monoclonal antibody medications such as Rituxan (rituximab) are medications that can aid in preventing your immune system from destroying platelets.
ProceduresIf medications are ineffective, certain procedures may be considered.
Blood or platelet transfusions are used for individual cases of heavy bleeding or among those at high risk of bleeding.
In some cases, surgical removal of the spleen, also called a splenectomy, may be recommended. The spleen stores platelets, and removing it can help improve your platelet levels in the bloodstream. However, this procedure carries potential risks, including bleeding, infection, and abnormal blood clots, so other treatment options may be offered first.
Reduce Your RiskWhile there aren't any ways to prevent thrombocytopenia, you can reduce your risk. Ask your healthcare provider about any medical conditions you have or prescribed medications you take that may increase your risk of thrombocytopenia. Work with them to create a treatment plan to reduce this risk, if possible.
Related ConditionsThrombocytopenia can be influenced by the following medical conditions:
Autoimmune disorders: These include immune thrombocytopenia (ITP), lupus, and rheumatoid arthritis, which can mistakenly trigger your immune system to attack and destroy platelets.
Cancer: This especially includes blood cancers such as leukemia or lymphoma. These types of cancers, and cancer treatments like radiation and chemotherapy, can damage your bone marrow and destroy blood stem cells.
Conditions that cause blood clots: These include thrombotic thrombocytopenic purpura (TTP) and disseminated intravascular coagulation (DIC).
Bacterial and viral infections: Thrombocytopenia can occur in chronic infections such as hepatitis B, hepatitis C, and human immunodeficiency virus (HIV).
An enlarged spleen: though rare, an enlarged spleen can be caused by certain conditions such as liver disease and leukemia.
Pregnancy: Mild thrombocytopenia can develop when you are close to delivery. It is possible to see a gradual decline in platelets from the first trimester until birth. This is attributed to the body's need to support the growing fetus.
Some surgical procedures: Platelets can be destroyed during surgical procedures involving artificial heart valves, blood vessel grafts, or the use of machines and tubing for blood transfusions or bypass surgery. This can occur as they pass through these artificial structures and equipment, leading to a decrease in platelet count.
The prognosis of thrombocytopenia varies and depends on the cause. The best way to prevent complications of thrombocytopenia is by working with your healthcare provider to establish a treatment regimen and determine what lifestyle changes you should make. Infection prevention measures should also be taken.
Tell your provider if you have any symptoms of an infection, such as a fever. Removing your spleen raises your risk of an infection. In this case, your provider may discuss taking steps to reduce your risk of infections, such as getting vaccinated. Monitor for symptoms of bleeding, which can quickly become a medical emergency.
If you are a smoker, then quitting smoking can make a huge difference in your health outcomes. Smoking can raise your risk of developing a blood clot.
Avoid contact sports, and ask your provider about physical activities that are safe for you. Take safety precautions, such as using a seatbelt while riding in a car and wearing gloves when working with knives and other tools.
Make sure to take all medicines as prescribed. Before any surgery or dental procedures, tell your healthcare provider about any medications you take that may lower your platelet count. These medicines may increase bleeding during these procedures. Avoid medicines that may lower your platelet count or stop your platelets from working properly. Aspirin and Advil (ibuprofen) are commonly used medicines that may thin your blood too much.
Frequently Asked QuestionsCan you live long with low platelets?
To manage low platelet levels and prevent complications, you should consult your healthcare provider. You may need routine testing to monitor your platelet levels and develop an appropriate treatment plan.
Is thrombocytopenia cancerous?
Thrombocytopenia itself is not cancerous, but it can be caused by cancers, like leukemia. It can also be caused by non-cancerous conditions, such as liver disease.
Is thrombocytopenia curable?
The treatment plan for thrombocytopenia varies based on the cause of this condition. If your platelet count is low because of a medication, for example, your healthcare provider may suggest switching to a different medication. Consult with your healthcare provider to identify what may be causing you to have thrombocytopenia.
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Argenx's ADVANCE-SC Study Of VYVGART Hytrulo In Primary ITP Fails To Meet Primary Or Secondary Endpoints
argenx SE, a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, announced topline results from the ADVANCE-SC study evaluating VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in adults with primary immune thrombocytopenia (ITP). The study did not meet the primary endpoint of a sustained platelet count response in chronic ITP patients.
Additional analyses of the dataset are ongoing and the full results will be presented at an upcoming medical meeting and in a peer-reviewed publication.
"This is not the outcome we had hoped for patients, but setbacks are part of pioneering a new class of medicines and these data will provide insights into the broader understanding of FcRn and ITP," said Luc Truyen, M.D., Ph.D., chief medical officer of argenx. "We are very grateful to everyone involved in the ADVANCE-SC study, especially the patients and their families, the investigators, and our internal team who worked tirelessly to complete this global study. We remain committed to the ITP patient community who urgently needs additional treatment options to manage this challenging disease, and continue to move forward in our deeper analysis of these results."
ADVANCE-SC is the second of two registrational trials conducted as part of the ongoing ITP development program for VYVGART and enrolled 207 adult patients with chronic and persistent ITP. Patients were heavily pre-treated and 75% of patients had received three or more prior ITP therapies.
Primary endpoint was not met (p=0.5081); 13.7% (17/124) of treated patients demonstrated a sustained platelet count response compared to 16.2% (11/68) of placebo patients.
Secondary endpoints were not met, including additional endpoints on International Working Group (IWG) responder status and mean platelet count change from baseline.
VYVGART Hytrulo was well-tolerated in ADVANCE-SC; the observed safety and tolerability profile was consistent with ADVANCE-IV and the confirmed safety profile of VYVGART and VYVGART Hytrulo.
Results from the first study in the ITP registrational programme, ADVANCE-IV, were reported in May 2022. The study met its primary and key platelet-derived secondary endpoints. ADVANCE-IV formed the basis of the regulatory submission for approval of VYVGART IV for ITP in Japan, where a decision is expected in the first quarter of 2024.
VYVGART is currently being evaluated in 13 severe autoimmune diseases, including the registrational ADDRESS study for pemphigus from which topline results are expected around year-end 2023.
The ADVANCE-SC trial was a randomized, double-blind, placebo-controlled, multicenter, global trial evaluating the efficacy and safety of VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in adult patients with chronic or persistent primary ITP. Enrolled patients had a confirmed ITP diagnosis and a mean entry platelet count of less than 30x109/L. Patients were on a stable dose of at least one ITP treatment prior to randomization and had received at least one prior therapy. Concomitant medications permitted included corticosteroids, nonsteroidal immunosuppressive drugs, fostamatinib or TPO-RAs. The study patients who were on 'watch and wait' at baseline were required to have received at least 2 prior treatments for ITP.
Patients were randomized in a 2:1 ratio to receive VYVGART Hytrulo or placebo for a total of 24 weeks as part of the primary trial. The primary endpoint was measured by the proportion of patients with chronic ITP with a sustained platelet count response defined as achieving platelet counts of greater than or equal to 50x109/L for at least four of the last six scheduled visits between weeks 19 and 24. Patients who received rescue therapy at week 12 or later, or for whom dose and/or frequency of concurrent ITP therapies increased at week 12 or later, were considered non-responders. Key secondary endpoints included extent of disease control over 24-week treatment period, proportion of overall population with sustained platelet count response, an extended primary endpoint analysis between weeks 17 and 24, and the incidence and severity of WHO-classified bleeding events.
Immune thrombocytopenia (ITP) is an autoimmune disorder where immunoglobulin G (IgG) autoantibodies destroy platelets and reduce platelet production, which can lead to an increased risk of excessive bleeding and bruising. In rare cases, ITP can lead to severe anaemia and life threatening gastrointestinal or intracranial haemorrhages. ITP is also associated with debilitating fatigue and significant impacts on mental health, including anxiety, fear and depression. Many ITP patients are inadequately controlled on current therapies so a significant unmet need exists for additional treatment options.
VYVGART Hytrulo is a subcutaneous combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE drug delivery technology to facilitate subcutaneous injection delivery of biologics. In binding to the neonatal Fc receptor (FcRn), VYVGART Hytrulo results in the reduction of circulating IgG.
VYVGART Hytrulo is the proprietary name in the US for subcutaneous efgartigimod alfa and recombinant human hyaluronidase PH20. It is marketed in Europe as VYVGART and may be marketed under different proprietary names following approval in other regions.
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines.
Fetomaternal Alloimmune Thrombocytopenia (FMAIT): Explained
This immune response can lead to low platelet levels in the fetus, causing bleeding and potential complications. While most cases are mild, severe instances can result in
.
, against fetal platelet antigens. Notably, there are 24 known human platelet-specific alloantigen (HPAs), with anti-HPA-1a being the most common in white populations. The timing and mechanism of immunization remain uncertain, but interactions between the maternal immune system and fetal cells, particularly trophoblasts, are considered crucial (
).
Recognizing FMAIT can be challenging, but suspicion arises if neonatal thrombocytopenia is incidentally discovered or if there's a family history of the condition. The clinical presentation varies based on the severity and onset of thrombocytopenia. Bleeding into the skin, often manifesting as petechiae or ecchymoses, is a common sign. Severe cases may involve bleeding into major organs, emphasizing the critical need for early diagnosis.Difference between Autoimmune and Alloimmune Thrombocytopenia Neonatal thrombocytopenia is relatively rare, and FMAIT stands out as the most common cause of severe cases. Distinguishing it from other conditions like infection, autoimmune thrombocytopenia, or genetic abnormalities is crucial for effective management. FMAIT differs from autoimmune thrombocytopenia as it exclusively affects fetal platelets due to maternal antibodies, leading to more severe manifestations.Advertisement
Autoimmune thrombocytopenia is caused by the immune system mistakenly recognizing platelets as foreign and producing antibodies against them. These antibodies then target and destroy platelets, leading to a low platelet count. This condition can occur in both children (immune thrombocytopenic purpura) and adults (immune thrombocytopenia) and is often idiopathic, meaning the exact cause is unknown.On the other hand, alloimmune thrombocytopenia occurs when a pregnant woman's immune system produces antibodies against her baby's platelets. This can happen if there is a mismatch between the mother's and baby's blood types, specifically in the ABO or Rh system. These antibodies can cross the placenta and destroy the baby's platelets, leading to thrombocytopenia in the newborn.
Challenges in Assessing FMAIT's Impact Assessing the severity of FMAIT poses challenges, and currently, there is no definitive maternal laboratory marker for predicting outcomes. A history of a previously affected child remains the most robust predictor of recurrence and severity. Maternal antibody titers may not consistently correlate with clinical severity, emphasizing the need for close monitoring and individualized assessments.While universal screening for FMAIT is not currently recommended, various approaches have been suggested to identify pregnancies at risk. Screening strategies involve measuring circulating anti-HPA antibodies or using genotyping techniques. However, the lack of a simple, safe, and validated screening test complicates the decision-making process.
Prenatal and Neonatal Management: Balancing Risks and Benefits Managing pregnancies at risk for FMAIT involves careful planning, with a focus on avoiding complications. Maternal intravenous immunoglobulin (IVIG), corticosteroids, or intrauterine platelet transfusions are potential treatments, each with its own set of considerations. Neonatal management centers around platelet transfusions, with the goal of maintaining adequate platelet levels and minimizing the risk of bleeding.FMAIT remains a complex and challenging condition that requires a multidisciplinary approach for effective management. While advancements in screening and treatment strategies continue, a nuanced understanding of the condition's intricacies is essential for improving outcomes and reducing the impact of this rare but serious pregnancy complication.
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