Thrombotic thrombocytopenic purpura (TTP)
Description
●The Introduction:
➣Thrombotic thrombocytopenic purpura (TTP) is defined as a thrombotic microangiopathy brought on by a lack of the enzyme ADAMTS13, which cleaves von Willebrand factor (VWF).
➣Types include:
• Acquired TTP: caused by autoantibody inhibitors, which are prevalent in adults.
• Upshaw-Schulman syndrome, or hereditary TTP, is caused by genetic alterations and is more prevalent in children.
➣Epidemiology:
• Adults: >95% acquired; 3 per million annually.
• Children: about 1 in 10 million annually (less than 18 years old).
➣Clinical impact: potentially fatal; around 34% of survivors relapse.
➣Options for treatment include immunosuppressants, plasma exchange (PEX), and the more recent use of caplacizumab, a nanobody that targets VWF.
➣Gap: No clinical trials or guidelines for children.
●Discussions
➣The diagnosis of TTP in children is difficult due to its rarity. must be distinguished from other TMAs and congenital TTP.
➣Survival increases from 20% to 80% with PEX. restores ADAMTS13 and eliminates antibodies.
➣Rituximab: Prevents relapses by reducing B-cells and inhibiting the creation of antibodies during the acute period.
➣VWF-platelet binding is blocked by caplacizumab, which is now authorised for use in adults and adolescents ≥12 years ≥40 kg.
• Reduces thrombotic events, PEX, hospital stays, and speeds up platelet recovery.
• Works best when paired with rituximab; does not address underlying immunological disease.
➣ Safety: There were no significant side effects or bleeding, and both patients handled caplacizumab well.
➣Implications for the future include:
• Potential PEX-free approaches including caplacizumab and immunosuppression;
• Additional paediatric studies are required.
Case 1: A sixteen-year-old girl
Syncope accompanied by a chin injury, fever, headache, nausea, renal impairment, severe anaemia, thrombocytopenia, schistocytes, elevated ESR, and CRP is shown.
• PLASMIC score = 7 (high risk) according to the workup.
ADMTS13 activity is less than 1%.
Antibodies against ADAMTS13: 66.9 U/ml.
Treatment: IVIG + LMWH + PEX + corticosteroids.
On day four, caplacizumab was administered.
• Four doses of rituximab while ADAMTS13 was still low.
➣Result: ADAMTS13 increased to 64% and platelets returned to normal in 10 days. Follow-up at 77 weeks: stable remission.
Protocol
●Paediatric TTP Patients' Main Issues:
➣ Severe thrombocytopenia (extremely low platelet count);
➣ Coombs-negative haemolytic anaemia (red blood cell destruction);
➣ Renal impairment (kidney dysfunction);
➣ Neurological symptoms (e.g., syncope, confusion, dysarthria);
➣ High anti-ADAMTS13 antibody titers (autoimmune reaction);
➣ Risk of bleeding, organ damage, and quick clinical decline
● Comprehensive Nursing Interventions: Issue and Resolution
Vario Thrombocytopenia Severe
• Issue: Patients with critically low platelet counts (less than 10 × 10⁹/L) are at immediate danger of organ damage and spontaneous bleeding.
• Handle the situation by keeping an eye out for any indications of bleeding, such as petechiae, ecchymosis, or mucosal bleeding.
→Take measures to prevent bleeding by avoiding invasive procedures and intramuscular injections, as well as addressing environmental dangers such as sharp objects.
→Support the start and ongoing monitoring of caplacizumab therapy and plasma exchange (PEX), which has been demonstrated to quickly recover platelet counts. Prepare and give drugs (corticosteroids, caplacizumab) in accordance with protocol; keep an eye out for any reactions.
➣Haemolytic Anaemia
• Issue: Fatigue, pallor, jaundice, increased LDH, and low haemoglobin levels were all signs of the sudden breakdown of red blood cells.
•Management: Keep an eye on lab results (reticulocyte count, haemoglobin, bilirubin, and LDH) and check for anaemia symptoms (fatigue, pallor, and tachycardia).
In the event of severe anaemia or cardiac compromise, prepare for and support a blood transfusion. →Keep an eye on oxygen saturation and administer extra oxygen as necessary.
➣ Damage to the kidneys
• Issue: Acute kidney damage can develop from mild to severe renal failure and elevated creatinine.
•Management: Keep a careful eye on electrolytes, serum creatinine, urine intake, and output.
Make sure that medications are administered in a way that is friendly to the kidneys by avoiding nephrotoxic drugs and modifying dosages as necessary.
Encourage hydration while preventing fluid excess; maintain meticulous records of fluid balance.
➣Neurological Signs and Symptoms
• Issue: Confusion, syncope, headaches, or dysarthria are symptoms that indicate cerebral microvascular involvement and stroke risk.
In order to prevent injuries during moments of confusion or weakness, safety precautions should be put in place. Frequent neurological examinations should be performed to identify any changes in consciousness or new deficiencies. New results should be promptly reported to the multidisciplinary team for immediate management.
➣ Elevated Autoimmune Activity Anti-ADAMTS13 Antibodies
• Issue: Immunosuppression is necessary because autoimmune generation of anti-ADAMTS13 antibodies prolongs disease activity.
• Management: →Keep an eye on and assist with the use of immunosuppressants (IVIG, rituximab, and high-dose corticosteroids).
Keep an eye out for and report any fever, localised symptoms, or abnormal lab results that may indicate an infection (caused by immunosuppression).
→Teach infection risk reduction techniques, such as mask wearing, hand cleanliness, and visitor screening.
➣ Assistance During PEX (plasma exchange)
• Issue: PEX is resource-intensive, technically demanding, and prone to problems (e.g., electrolyte imbalance, allergic response, and hypovolemia).
• Management: → Set up and prime equipment; confirm procedure and identity.
During the procedure, keep an eye on vital signs and the access site (for hypotension, haemorrhage, or infection). If necessary, check calcium levels and give prophylactic calcium.
➣Planning for Psychosocial, Educational, and Discharge
•Anxiety, uncertainty, and the practical need for home care are exacerbated by the chronic and recurring nature of TTP and intense therapy.
• Management: Explain the condition, treatments (including off-label treatments like caplacizumab), and objectives. Assist the family with transportation and follow-up, including teaching them how to administer caplacizumab at home according to a specialist's protocol. Check for psychological distress, provide resources for counselling, and organise interdisciplinary support.
Notes
For more details 10.3389/fped.2021.743206
