The Global Thalassemia Market report provides a comprehensive study of global thalassemia market and also major regional markets. For more mail: vikas@konceptanalytics.com

1. Global Thalassemia Market Report
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2016
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2. Executive Summary
 Thalassemia is a hereditary blood disorder in which a person’s body makes an abnormal form of hemoglobin,
which is the protein molecule in red blood cells (RBCs), which carries oxygen. The disorder results in
unwarranted damage of red blood cells, which leads to anemia. Anemia is a condition in which the body doesn’t
have adequate amount of normal and healthy red blood cells. There are majorly two types of thalassemia based
on these proteins known as the Alpha thalassemia and the Beta thalassemia. There is also a third type viz.
Sickle beta thalassemia, which is like a combination of sickle cell disease and beta thalassemia.
 Even though it is a rare genetic disorder, the number of people affected by it has been growing with time. While
those with thalassemia minor have hardly any threat to their life and only require occasional blood transfusion to
sustain, thalassemia major patients need to go for periodic transfusions for a sustained life. In case of infants,
thalassemia major often leads to still birth or cause death shortly after the baby’s birth. Presently the only cure for
thalassemia is a bone marrow transplantation therapy, which is done mostly for severe cases of thalassemia.
 The key factors which are anticipated to drive this market include rising healthcare expenditure, rising spending
on stem cell therapy research and development and rising population in the East-Asia and Pacific region of the
world. Some of the noteworthy progresses of this industry include the advances in the iron chelation therapy,
development of gene therapy and fetal hemoglobin inducers as potential curatives for thalassemia. However, the
growth of respective industry is challenged by the high cost of treatments, lack of alternative treatments and
unmet market of the Asia-Pacific.
 The report provides a comprehensive study of global thalassemia market and also major regional markets.
Furthermore, market dynamics such as key trends and development; and challenges are analyzed in depth. The
global thalassemia industry is highly competitive consisting of several companies including the Lonza group,
Acceleron Pharma, BlueBird Bio and Sangamo Biosciences etc. The competitive landscape of the respective
market, along with the company profiles of the leading players are also discussed in detail.
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3. Bone marrow transplantation is presently the only chief cure for thalassemia major patients or patients
with transfusion-dependent thalassemia
• It is noted that out of the total people suffering from severe
thalassemia, only xx% cases are estimated to be
diagnosed with thalassemia major non-β0/β0 type, while
xx% cases are diagnosed with phenotypical β0/β0 type of
thalassemia.
• It is estimated that in the year 2015, nearly xx.xx% of the
global transfusion-dependent β thalassemia patients were
from the Middle-East region and xx.x% were from the
South-East Asian region.
• The global bone marrow transplant therapy revenue is
estimated to be US$xx.xx billion for the year 2015. It is
estimated to grow at a CAGR of xx% over the next five
years and is forecasted to reach US$xxx by the end of the
year 2020.
: Global Transfusion-Dependent β Thalassemia by Region (2015E)
Global Thalassemia Diagnosis by Category (2015E)
Global Bone Marrow Transplant Revenue (2015E-2020F)
Non-Severe
Major Non-
β0/β0
Phenotypical
β0/ β0
Severe
Europe Americas SouthEast Asia Middle East Pacific region Africa
2015E 2016F 2017F 2018F 2019F 2020F
US$Billion

4. Transfusion-dependent patients are those whose level of severity of disease is so high that regular blood
transfusions have to be conducted for a sustained life and healthy functioning of these patients.
• The number of beta thalassemia carriers in the US is estimated to
have reached …. thousand in the year 2015. The number is expected
to grow further in the future years, and is forecasted to grow at a
CAGR of xx% over the period 2016-2020
• The number of beta thalassemia major patients for the year 2014 in the
US is noted to be xx.x thousand and is estimated to have reached xx
thousand in the year 2015, marking a slight growth of xx%.
• It is forecasted that the number of transfusion-dependent β
thalassemia patients in the US will rise gradually over time, reaching a
total of xx thousand patients by the end of the year 2020.
The US β Thalassemia Major Patients – Forecast (2016F-2020F)
The US β Thalassemia Carrier Patients – Forecast (2016F-2020F)
The US Transfusion-Dependent β Thalassemia Patients (2016F-2020F)
2016F 2017F 2018F 2019F 2020F
ThousandPersons
2016F 2017F 2018F 2019F 2020F
ThousandPersons
2016F 2017F 2018F 2019F 2020F
ThousandPersons

5. The most effective type and also the most hard to find is the sibling-matched bone marrow transplant.
• It is forecasted that the number of patients taking sibling-
matched stem cell transplant among β thalassemia patients in
the US will reach a sum of xx0 thousand persons by the year
2020.
• It is forecasted that the number of transfusion-dependent β
thalassemia patients in the Europe will increase further at a
CAGR of xx% over the period 2016-2020, thereby reaching a
sum of xxx thousand patients by the end of the year 2020.
• Out of the total number of transfusion-dependent β thalassemia
patients in the Europe, it is estimated that nearly xxx thousand
patients are those with major/intermediate level of severity, in the
year 2015.
Europe Transfusion-Dependent β Thalassemia Patients
The US β Thalassemia Sibling-Matched Stem Cell Transplant (2016F-2020F)
Europe Transfusion-Dependent β Thalassemia Major Patients
2014 2015E 2016F 2017F 2018F 2019F 2020F
ThousandPersons
2015E 2016F 2017F 2018F 2019F 2020F
ThousandPersons
2016F 2017F 2018F 2019F 2020F
ThousandPersons

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