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aTTP/iTTP is a medical emergency and early diagnosis is critical1,2

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Acquired/immune-mediated thrombotic thrombocytopenic purpura (aTTP/iTTP) is a rare, rapidly progressing, life-threatening medical emergency. It has been reported in literature that, when left untreated, death may occur in up to 90% of patients with aTTP/iTTP, making urgent diagnosis and treatment a necessity.1-6

Identifying aTTP/iTTP is crucial for initiation of an appropriate therapeutic strategy7

Diagnose aTTP/iTTP through clinical assessment or risk assessment tools prior to ADAMTS13 testing.

Clinical assessment7

OR

Risk assessment7*

Patient presentation prompting suspicion of aTTP:

  • Thrombocytopenia (<100 × 109/L)
  • Evidence of microangiopathic hemolytic anemia
    • Hb and hematocrit below reference range
    • Low haptoglobin
    • Elevated LDH
    • Presence of schistocytes in peripheral blood smear
  • Relatively preserved renal function

Available risk assessment tools include:

  • PLASMIC score
  • French score

The higher the risk assessment score, the more likely patients have severe ADAMTS13 deficiency and aTTP/iTTP

*ISTH did not appraise the evidence for these 2 tools.

Risk assessment tools can be used to predict ADAMTS13 deficiency

table
table

Complex presentation of TTP often causes a delay in diagnosis10

TTP presents with highly variable, multiorgan symptoms that resemble a series of thrombotic microangiopathy (TMAs) and other disorders. It is therefore critical to differentiate TTP from other similarly presenting conditions. The table below compares TTP with other resembling conditions.

Differentiating aTTP/iTTP from other similarly presenting TMAs11-18

 TTPHUSDIC
Presenting symptoms

• Thrombocytopenia (platelet count <150 × 109/L or >25% reduction from baseline)11,12
• MAHA (presence of schistocytes)11,12
• Organ ischemia11,12

TypesCongenital and acquired/immune-mediated TTP (cTTP and aTTP/iTTP)13,14Infection-associated or atypical/complement-mediated HUS (IA-HUS or aHUS/CM-HUS)14,15Overt and nonovert DIC16
AgecTTP—usually
children14
aTTP/iTTP—usually adults14		IA-HUS—common in
children17
aHUS/CM-HUS—any age17		NA
CausecTTP—inherited mutations of ADAMTS1314
aTTP/iTTP—autoantibodies against ADAMTS1314		IA-HUS—toxins produced by certain bacteria17,18
aHUS/CM-HUS—activation of the complement system17,18		Occurs as a result of underlying diseases such as sepsis, malignancy, trauma, liver diseases, obstetric disorders, envenomation, vascular anomalies, and major transfusion reactions16
Potential laboratory resultsPlatelet count <30 × 109/L18
Creatinine <2.25 mg/dL18
PT and aPTT—normal11
D-dimer—normal11
ADAMTS13 <10%18		Platelet count <30 × 109/L18
Creatinine >2.25 mg/dL18
PT and aPTT—normal11
D-dimer—normal11
ADAMTS13 ≥10%18		Platelet count <50 × 109/L17
PT and aPTT—prolonged16,17
D-dimer—elevated16,17
Test(s) confirming the diagnosiscTTP—ADAMTS13 sequencing14,18

aTTP/iTTP—ADAMTS13 testing14,18		IA-HUS—culture test, PCR, ADAMTS13 testing11,17

aHUS/CM-HUS—culture test, ADAMTS13 testing11,17		ISTH scoring system for overt DIC16

Severe thrombocytopenia, MAHA, and organ ischemia indicate aTTP/iTTP is likely13

See what Guidelines suggest about treating as soon as you suspect aTTP/iTTP.

Is it TTP or aHUS?

The clinical presentation of aTTP/iTTP can be strikingly similar to that of aHUS, which is why diagnosis can often be complicated. The creatinine levels, platelet count, and ADAMTS13 levels may guide toward distinguishing one from the other.13,14

Differentiating TTP from HUS is crucial in order to start an appropriate therapy14

The following chart can help guide diagnosis to quickly differentiate patients with TTP in need of emergency care.

Flowchart for differentiating TTP from HUS and other TMAs

Adapted from: Kremer Hovinga JA et al. Nat Rev Dis Primers. 2017;3:17020.

Biopsies can be useful in differentiating aTTP/iTTP from aHUS in difficult cases19

Biopsies can be useful in difficult diagnostic situations. Sampling of any accessible, highly vascular site may help inform the differential diagnosis of a TMA, based on pathologic distinctions between aTTP/iTTP and aHUS.

Icon of platelets with vWF

aTTP/iTTP

Biopsy sample of any accessible, highly vascular site shows the following:

 

  • Microthrombi appear as “white clots” composed of platelets and vWF, with only small amounts of fibrin

 

  • Vascular or perivascular inflammatory cell infiltrations are minimal or absent
Icon of platelets with fibrin

aHUS/CM-HUS

Biopsy sample of any accessible, highly vascular site shows the following:

 

  • Microthrombi appear as “red clots,” predominated by fibrin

 

  • An inflammatory infiltrate may be seen together with deposits of C5b-9

EHR capabilities such as Rule Messages can support proactive identification of at-risk patients for further evaluation to differentiate aTTP/iTTP from other conditions

DOWNLOAD THE EHR GUIDE

Arterial thrombosis icon

aTTP/iTTP presents similarly to other TMAs

DIFFERENTIATE aTTP/iTTP
Guidelines checkmarks

ISTH Guideline recommendations

SEE THE RECOMMENDATIONS

ADAMTS13=a disintegrin and metalloproteinase with a thrombospondin type 1 motif, 13; aHUS=atypical hemolytic uremic syndrome; aPTT=activated partial thromboplastin time; CM-HUS=complement-mediated hemolytic uremic syndrome; cTTP=congenital thrombotic thrombocytopenic purpura; DIC=disseminated intravascular coagulation; EHR=electronic health record; Hb=hemoglobin; HUS=hemolytic uremic syndrome; IA-HUS=infection-associated hemolytic uremic syndrome; ISTH=International Society on Thrombosis and Haemostasis; LDH=lactate dehydrogenase; MAHA=microangiopathic hemolytic anemia; MCV=mean corpuscular volume; PCR=polymerase chain reaction; PT=prothrombin time; TTP=thrombotic thrombocytopenic purpura; vWF=von Willebrand factor.

INDICATION

CABLIVI (caplacizumab-yhdp) is indicated for the treatment of adult and pediatric patients 12 years of age and older with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS: 
CABLIVI is contraindicated in patients with previous severe hypersensitivity reaction to caplacizumab-yhdp or to any of its excipients. Hypersensitivity reactions have included urticaria.

WARNINGS AND PRECAUTIONS:
Hemorrhage:

  • CABLIVI increases the risk of bleeding. In clinical studies, severe bleeding adverse reactions of epistaxis, gingival bleeding, upper gastrointestinal hemorrhage, and metrorrhagia were each reported in 1% of subjects. Overall, bleeding events occurred in approximately 58% of patients on CABLIVI versus 43% of patients on placebo.
  • In the postmarketing setting cases of life-threatening and fatal bleeding were reported in patients receiving CABLIVI.
  • The risk of bleeding is increased in patients with underlying coagulopathies (e.g. hemophilia, other coagulation factor deficiencies). It is also increased with concomitant use of CABLIVI with drugs affecting hemostasis and coagulation.
  • Avoid concomitant use of CABLIVI with antiplatelet agents, thrombolytic drugs, heparin or anticoagulants. If clinically significant bleeding occurs, interrupt use of CABLIVI. Von Willebrand factor concentrate may be administered to rapidly correct hemostasis. If CABLIVI is restarted, monitor closely for signs of bleeding.
  • Withhold CABLIVI for 7 days prior to elective surgery, dental procedures or other invasive interventions. If emergency surgery is needed, the use of von Willebrand factor concentrate may be considered to correct hemostasis. After the risk of surgical bleeding has resolved, and CABLIVI is resumed, monitor closely for signs of bleeding.

ADVERSE REACTIONS:
In adults, the most common adverse reactions (>15% of patients) are epistaxis, headache, and gingival bleeding. In pediatric patients, the most frequently reported adverse reactions are epistaxis and tachycardia.

DRUG INTERACTIONS:
Concomitant use of CABLIVI with any anticoagulant or antiplatelet agent may increase the risk of bleeding. Avoid concomitant use when possible. Assess and monitor closely for bleeding with concomitant use.

PREGNANCY:
There are no available data on CABLIVI use in pregnant women to inform a drug associated risk of major birth defects and miscarriage.

  • Fetal/neonatal adverse reactions: CABLIVI may increase the risk of bleeding in the fetus and neonate. Monitor neonates for bleeding.
  • Maternal adverse reactions: All patients receiving CABLIVI, including pregnant women, are at risk for bleeding. Pregnant women receiving CABLIVI should be carefully monitored for evidence of excessive bleeding.

Please see full Prescribing Information.
Instructions For Use
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INDICATION

IMPORTANT SAFETY INFORMATION

References: 1. Scully M, Hunt BJ, Benjamin S, et al; British Committee for Standards in Haematology. Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br J Haematol. 2012;158(3):323-335. doi:10.1111/j.1365-2141.2012.09167.x 2. Goel R, King KE, Takemoto CM, Ness PM, Tobian AAR. Prognostic risk-stratified score for predicting mortality in hospitalized patients with thrombotic thrombocytopenic purpura: nationally representative data from 2007 to 2012. Transfusion. 2016;56(6):1451-1458. doi:10.1111/trf.13586 3. Grall M, Azoulay E, Galicier L, et al. Thrombotic thrombocytopenic purpura misdiagnosed as autoimmune cytopenia: causes of diagnostic errors and consequence on outcome. Experience of the French Thrombotic Microangiopathies Reference Centre. Am J Hematol. 2017;92(4):381-387. doi:10.1002/ajh.24665 4. Kremer Hovinga JA, Vesely SK, Terrell DR, Lämmle B, George JN. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2010;115(8):1500-1511. doi:10.1182/blood-2009-09-243790 5. Peyvandi F, Scully M, Kremer Hovinga JA, et al. Caplacizumab reduces the frequency of major thromboembolic events, exacerbations and death in patients with acquired thrombotic thrombocytopenic purpura. J Thromb Haemost. 2017;15(7):1448-1452. doi:10.1111/jth.13716 6. Sayani FA, Abrams CS. How I treat refractory thrombotic thrombocytopenic purpura. Blood. 2015;125(25):3860-3867. doi:10.1182/blood-2014-11-551580 7. Zheng XL, Vesely SK, Cataland SR, et al. ISTH guidelines for the diagnosis of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2020;18(10):2486-2495. doi:10.1111/jth.15006 8. Coppo P, Cuker A, George JN. Thrombotic thrombocytopenic purpura: toward targeted therapy and precision medicine. Res Pract Thromb Haemost. 2018;3(1):26-37. doi:10.1002/rth2.12160 9. Bendapudi PK, Hurwitz S, Fry A, et al. Derivation and external validation of the PLASMIC score for rapid assessment of adults with thrombotic microangiopathies: a cohort study. Lancet Haematol. 2017;4(4):e157-e164. doi:10.1016/S2352-3026(17)30026-1 10. Gallan AJ, Chang A. A new paradigm for renal thrombotic microangiopathy. Semin Diagn Pathol. 2020;37(3):121-126. doi:10.1053/j.semdp.2020.01.002 11. Vincent J-L, Castro P, Hunt BJ, et al. Thrombocytopenia in the ICU: disseminated intravascular coagulation and thrombotic microangiopathies—what intensivists need to know. Crit Care. 2018;22(1):158. doi:10.1186/s13054-018-2073-2 12. Nguyen TC, Cruz MA, Carcillo JA. Thrombocytopenia-associated multiple organ failure and acute kidney injury. Crit Care Clin. 2015;31(4):661-674. doi:10.1016/j.ccc.2015.06.004 13. Joly BS, Coppo P, Veyradier A. Thrombotic thrombocytopenic purpura. Blood. 2017;129(21):2836-2846. doi:10.1182/blood-2016-10-709857 14. Chiasakul T, Cuker A. Clinical and laboratory diagnosis of TTP: an integrated approach. Hematology Am Soc Hematol Educ Program. 2018;2018(1):530-538. doi:10.1182/asheducation-2018.1.530 15. Wada H, Matsumoto T, Suzuki K, et al. Differences and similarities between disseminated intravascular coagulation and thrombotic microangiopathy. Throm J. 2018;16:14. doi:10.1186/s12959-018-0168-2 16. Venugopal A. Disseminated intravascular coagulation. Indian J Anaesth. 2014;58(5):603-608. doi:10.4103/0019-5049.144666 17. Canpolat N. Hemolytic uremic syndrome. Turk Pediatri Ars. 2015;50(2):73-82. doi:10.5152/tpa.2015.2297 18. Kremer Hovinga JA, Coppo P, Lämmle B, Moake JL, Miyata T, Vanhoorelbeke K. Thrombotic thrombocytopenic purpura. Nat Rev Dis Primers. 2017;3:17020. doi:10.1038/nrdp.2017.20 19. Laurence J, Haller H, Mannucci PM, Nangaku M, Praga M, de Cordoba SR. Atypical hemolytic uremic syndrome (aHUS): essential aspects of an accurate diagnosis. Clin Adv Hematol Oncol. 2016;14(11)(suppl 11):2-15.

CABLIVI and Sanofi are registered trademarks of Sanofi or an affiliate. MAT-US-2108056-v6.0-01/2026 Last Updated: January 2026.

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