Congenital amegakaryocytic thrombocytopenia (CAMT) is an inherited and rare disorder. The main character of CAMT is severely decreasing megakaryocytes number. Megakaryocytes are kind of bone marrow cells that constitute the platelets.
Platelets play a role in blood clotting and thus prevent bleeding. In initial phase of CAMT, the bone marrow stops producing platelets and as the disease progresses it can stop making RBCs and WBCs also.
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Following are the symptoms of CAMT are: excessive bleeding and bruising and petechial like tiny red dots on skin. The primary cause of CAMT is a mutation in the gene for c-Mpl receptor that binds thrombopoeitin (TPO) for proper functioning.
This disorder is commonly seen in children. Children with CAMT may develop central nervous system abnormalities, lack of psychomotor development and also cardiac defects.
One of the very limited treatment option is Bone Marrow/Stem Cell Transplant. Research is carried out for obtaining TPO mimetics that can bind the c-Mpl receptor. Around 30% deaths in CAMT result due to excessive bleeding.
Increasing prevalence of rare diseases due to changing lifestyle and increasing pediatric diseases is the major factor contributing for the growth of congenital amegakaryocytic thrombocytopenia (CAMT) market.
Increasing rate of hematopoietic disorders like CAMT and improving success rates of stem cell transplantation can be driving forces of the CAMT market. Treatment options for CAMT are very less and lack of awareness of rare diseases in under developed regions of the world make the congenital amegakaryocytic thrombocytopenia (CAMT) market challenging.
The treatment costs for stem cell transplantation and bone marrow transplantation are extremely high. Drug development is advancing for rare diseases but still there 7000 rare diseases that do not have any treatment options.
Making it a major setback for the congenital amegakaryocytic thrombocytopenia (CAMT) market growth. The stem cell transplantation might not be successful and it’s considered that around 20% of deaths in CAMT result due to Hematopoietic stem cells transplant.
In the last few decades the Hematopoietic stem cells transplantation market has been rapidly growing which can be a boon for CAMT patients and thus CAMT market.
The global congenital amegakaryocytic thrombocytopenia (CAMT) Market is expected to grow in the next decade. Increasing adoption of stem cell transplantation is a major opportunistic factor for the global congenital amegakaryocytic thrombocytopenia (CAMT) Market players.
Based on type treatment, the stem cell transplant is expected to be the most lucrative segment due to growing stem cell research and adoption of stem cell transplantation. Based on route of administration, Intravenous Infusion is going to be most used method of modality as it is commonly used to transfer stem cells in the blood stream.
The TPO mimetics are used in many chemotherapy treatments and will see a growing rise in congenital amegakaryocytic thrombocytopenia (CAMT) Market as well.
With increasing extensive research and development activities in North America, Congenital amegakaryocytic thrombocytopenia (CAMT) Market is expected to hold a significant share of the market.
Majority of the research and development activities are carried out by key players based out of the US and UK. Asia Pacific countries like Japan, China, India and Australia have shown growth in number stem cell transplants in the recent years. The healthcare system is also improving in these regions.
Some of the key players in the Congenital amegakaryocytic thrombocytopenia (CAMT) market include
- Amgen Inc
- Intas Pharmaceuticals Ltd.
- Kyowa Kirin Co.
- Ltd GlaxoSmithKline plc.
- Novartis AG
- Lonza
- CBR Systems, Inc
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