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RECOGNIZING
TYPE 1 GAUCHER DISEASE

Type 1 Gaucher disease is a rare, autosomal recessive, chronic, progressive disorder caused by deficient activity of the lysosomal enzyme glucocerebrosidase1–3
Female patient hero blob
PREVALENCE

Although rare, Gaucher disease is the most common type of lysosomal storage disease.3 Type 1 is the most common form of Gaucher, with a higher prevalence in the Ashkenazi Jewish population, affecting1,3:

~1–9 IN 100,000

within the overall population4

~1 IN 600

within the Ashkenazi Jewish population5–7

~1 IN 17

within the Ashkenazi Jewish community that are carriers8

COMMON SIGNS & SYMPTOMS OF TYPE 1 GAUCHER DISEASE

Gaucher disease is a multi-systemic and progressive disease.1,3 Symptom onset can range from early childhood into late adulthood.9 Signs and symptoms may vary from patient to patient.3 It is important to be aware of the possible combinations of signs and symptoms, as this can help lead to earlier diagnosis.

LOW HEMOGLOBIN CONCENTRATION1

Hemoglobin

ANEMIA & FATIGUE1,3,10

LOW PLATELET COUNT1,3,10

Platelets

BRUISING & BLEEDING1,3,10

ENLARGED
SPLEEN1,3,10

Damaged Spleen

ABDOMINAL DISTENSION & DISCOMFORT1,3,10

ENLARGED
LIVER1,3,10

Enlarged Liver
A combination of these signs and symptoms may be indicative of type 1 Gaucher disease.
If you suspect type 1 Gaucher disease, test now.
Male patient
DIAGNOSTIC CHALLENGES

Accurate and timely diagnosis is important for the management of type 1 Gaucher disease.11

    The diagnosis of type 1 Gaucher disease often occurs several years after the first onset of clinical signs, and patients may experience diagnostic delays of up to 10 years.12,13 This could be due to several factors:

  • As a rare disease, the overall awareness of Gaucher disease is limited13
  • The clinical manifestations are highly heterogeneous, both in type and severity, presenting at a wide range of ages13,14
  • The earliest symptoms are general and non-specific so may be difficult to recognize14
  • Patients may be misdiagnosed and referred to multiple specialists before receiving an accurate diagnosis13,14
Delayed diagnosis can have serious medical consequences, including the development of potentially irreversible complications.13
TESTING FOR TYPE 1 GAUCHER DISEASE

    Type 1 Gaucher disease can be confirmed with:

  • β-Glucosidase enzyme assay — the gold standard of diagnosing type 1 Gaucher disease is a blood test that measures levels of glucosidase activity10,13
  • Genetic testing — identifies Gaucher disease-causing gene mutations and carrier status5,12
VPRIV HCP Brochure

Summary of VPRIV, an ERT for type 1 Gaucher disease

IMPORTANT SAFETY INFORMATION

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.