Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare blood
disease of bone marrow stem cells, which are genetically
characterized by the somatic mutation in the phosphatidylinositol
glycan protein A (PIG-A) gene. PNH generally occurs in the early 30s.
Around 10% patients develop PNH symptoms at 21 years of age or
earlier. Around 1 to 5 individuals per million people in the U.S. are
estimated to suffer from PNH. This is much lower than the incidence
rate of bone marrow aplasia. PNH often goes unrecognized; delay in
diagnosis may range from one year to more than 10 years.
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The global PNH treatment market is anticipated to expand at a rapid
pace during the forecast period. It is a niche market, with many
pharmaceutical and biotech companies investing in research of bone
marrow stem cells. According to current studies, the ideal treatment
available is to replace all the hematopoietic stem cells with normal
stem cells via stem cells transplantation. However, this treatment is
not ideal in some cases as stem cell transplantation requires a
stable histocompatible donor.
Complete stem cells transplantation is usually considered in severe
cases of PNH, for instance aplastic anemia and transformation to
leukemia, as these can be life threatening complications. Factors
driving the PNH treatment market include rise in number of blood &
bone marrow related disorders, increase in aging population, and
technological advancements in stem cells transplantation. However,
increase in cost of medical equipment, specifically surgical
equipment required for stem cell transformation; lack of
reimbursement policies in developing regions; and occurrence of side
effects in related current available treatments may hamper the PNH
treatment market.
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The global PNH treatment market can be segmented based on diagnosis
test, type of treatment, drugs, and end-user. In terms of diagnosis
test, the market can be divided into complete blood count test (CBC),
lactate dehydrogenase test (LDH), bilirubin test, bone marrow
examination, urine test for hemosiderin, flow cytometry, and others.
Based on the type of treatment, the PNH treatment market can be
segregated into treatment of PNH patients associated with hemolysis,
treatment of PNH patients associated with thrombosis, treatment of
PNH patients associated with non-hemolytic anemia, allogeneic stem
cell transplant (SCT)/bone marrow transplant (BMT), treatment of
pregnant PNH patients, treatment of pediatric PNH patients, and
others. In terms of drugs, the market can be classified into
eculizumab (Soliris), ALXN1210, and others. Based on end-user, the
PNH treatment market can be split into hospitals, pharmaceutical &
biotech companies, clinics, academic & research institutes, and
others.
Geographically, the market for PNH treatment can be divided into
North America, Europe, Asia Pacific, Latin America, and Middle East &
Africa (MEA). North America dominates the global PNH treatment market
due to the rise in the number of blood & bone marrow related
diseases, availability of satisfactory reimbursement policies, and
increase in awareness about the early diagnosis of the disease in the
region. The market in Europe is also expected to expand rapidly, as
key players are collaborating with research institutions and labs to
develop new innovative products. The PNH treatment market in Asia
Pacific is anticipated to expand at a fast pace owing to the unmet
needs regarding PNH treatment of the growing population.
Additionally, factors such as development of the health care network,
rise in disposable income, increase in health care awareness, and
availability of reimbursement facilities are boosting the PNH
treatment market in Asia Pacific.
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Key players operating in the PNH treatment market include Alexion
Pharmaceuticals, Inc., Thermo Fisher Scientific Inc., GE Healthcare,
and Johnson & Johnson.
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