Sickle cell disease treatment

Sickle cell disease is a blood disorder that is rampant in sub-Saharan Africa. Sickle cell disease also called sickle cell anemia is a genetic blood disorder. In Sickle cell disease, the red blood cells are distorted into a sickle shape or moon-crescent shape. Moreover, in sickle cell disease, some of the red blood cells become rigid and sticky and may form a clump and reduce or block the smooth blood flow. Hypoxia may cause pain crisis, a stroke, and infections due to necrosis of oxygen-deprived blood cells. Treatments of sickle cell disease may involve medications to relieve pain crises and symptoms, frequent blood transfusions, and a curative treatment - an allogeneic bone marrow transplant. We offer low-cost successful medical treatment (a bone marrow transplant) for cure of the sickle cell disease.
Sickle cell disease treatment
Sickle cell disease or sickle cell anemia is an inherited blood disorder. A child is born with sickle cell disease because of the passing of faulty genes from both parents to the child. The gene responsible for developing Hemoglobulin is affected in sickle cell disease patients. When both the father and mother have sickle cell traits ( the affected gene controlling the production of hemoglobin) and pass the affected genes on to the child, the child will have sickle cell disease. If the child receives only one affected gene from either of the parents, he/she will have sickle cell trait, but when the child gets the affected gene from both parents ( Sickle cell trait carrier), he/she develops sickle cell disease. A child with sickle cell disease will not be able to thrive normally and will have frequent health issues. Some of the complications of sickle cell disease can be life-threatening in the long run without sickle cell disease treatments. We as global healthcare guardians for international patients seeking Sickle cell disease treatment in India provide all the medical tourism assistance to them. We assist them in getting specialized medical treatments for Sickle cell disease with the best sickle cell disease treatment doctors in India. The Top Sickle Cell Disease Treatment Hospitals in India offer low-cost specialized medical treatments for sickle cell disease. CMCS Health, the leading medical tourism assistance company has helped scores of sickle cell disease patients from sub-Saharan Africa in getting cures for sickle cell disease.
Video - Know about sickle cell disease and it's curative treatment.
Epidemiology of Sickle Cell Disease:
Sickle cell disease is more common in tropical regions, especially those where malaria was or is still rampant, such as sub-Saharan Africa, Spanish-speaking regions in the Western Hemisphere, Saudi Arabia, India, and Mediterranean countries. Sickle cell disease is widely spread in certain ethnicities like the people residing in sub-Saharan Africa or the people of African descent, the Hispanic people from South and Central America, the Caribbean, and people of Middle Eastern, Asian, Indian, and Mediterranean descent. The disease has spread to many other regions and countries because of population migrations. The prevalence of sickle cell disease can be gauged from the following published data in various studies:
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- Currently, more than 4.4 million people have sickle cell disease, and almost 43 million people are carriers of sickle cell trait worldwide.
- The spread of sickle cell disease is highest in Sub-Saharan African countries and accounts for almost 80% of sickle cell disease cases worldwide.
- People from Nigeria are affected most by sickle cell disease among all African countries.
- More than 1'00'000 people are affected with sickle cell disease in The United States.
- Each year more than 90'000 children are born in Nigeria with Sickle cell trait.
- Each year around 40'000 sickle cell children are born in the Democratic Republic of Congo.
- Kenya also has almost 14000 sickle cell children born annually.
- Around 12'000 sickle cell children are born in Tanzania annually
- Every year 11'000 children with sickle cell trait are born in Uganda
Sickle cell disease is also rampant in other Sub-Saharan African countries like Angola, Cameroon, Zambia, Kenya, Ghana, Niger and many other African countries. As a Global Medical tourism assistance company, we assist international patients seeking specialized medical treatments for sickle cell disease at the top Sickle cell treatment hospitals in the world.
Video - Sickle cell disease prevalence in Sub-Saharan Africa - CMCS Health.
The Etiology of Sickle Cell Disease:
Sickle cell disease manifests as the transformation of normal red blood cells which are circular, disc-shaped, and flexible into Sickle or crescent moon-shaped red blood cells that are brittle, rigid, and sticky. Normal red blood cells contain a protein called Hemoglobuloin. A mutation in a certain HBB gene affects the hemoglobulin and subsequently the red blood cells. A person may carry a mutation in the HBB gene affecting the hemoglobulin but still will not have the complications of sickle cell disease, such a person has a sickle cell trait and is called a sickle cell carrier. When a man and woman who both have sickle cell trait marry each other and both of them pass the mutated HBB genes affecting hemoglobulin to their child, the child with two mutated HBB genes ( One from each of the parents) will have sickle cell disease. If the child receives only one mutated HBB gene from either of the parents, he will not have sickle cell disease but will have sickle cell trait and a carrier of sickle cell disease.
If both the husband and wife have sickle cell trait, there is a 50% chance that one of their children may have sickle cell trait, if the child inherits the sickle cell gene from one of the parents. If one of the children of sickle cell carrier parents inherits two sickle cell genes (one each from both parents) there is a 25% chance that the child may have sickle cell disease.
How mutation in the Hb gene affects the Red Blood cells:
Hemoglobulin is a protein in the red blood cells that carries oxygen to the rest of the cells in the human body. The normal red blood cells are circular, disc-shaped, and flexible and can pass through the tiniest of the blood capillaries to deliver oxygen to all the cells of the body. A mutation in the HBB gene changes the nature of hemoglobulin as it becomes sticky and rigid and forms solid clumps that cause a change in the shape of red blood cells. The altered hemoglobulin transforms the normal red blood cells into sickle or crescent moon-shaped red blood cells. As the sickle cells can not navigate through the blood capillary network in the human body, the affected portion's cells may not get oxygen and may start dying (necrosis), and will cause episodes of pain (pain crises) and frequent infections in sickle cell disease patients.
Moreover, normal red blood cells follow a life cycle, they die after 120 days and are replaced by new red blood cells made in the bone marrow. The life span of sickle-shaped red blood cells is much shorter (10 - 20 days), so when the bone marrow is not able to replace the fast-drying red blood cells, there will be low red blood cells in the sickle cell patients. Low red blood cells mean low hemoglobulin and sickle cell patients may suffer from severe anemia.
Types of Sickle Cell Disease:
Sickle cell disease is not a single disease but a group of hemoglobinopathies. It depends on the type of HBB gene that is mutated and inherited by the patient from the parents. The types of sickle cell disease depend on mutations in the gene that encodes the beta subunit of hemoglobin:
Hemoglobin SS (HbSS):
HbSS gene mutation causes the most common and severe sickle cell disease. Almost 65% of all sickle cell disease patients have this type of Hb gene mutation. Children with sickle cell disease inherit one gene from both parents encoded with HbS. In patients with this type of SCD, total hemoglobin is abnormal and patients suffer from severe anemia.
Hemoglobin SC (HbSC):
In this type of Sickle cell disease patients, the children inherit one mutated HbS gene from one parent and another abnormal Hb Gene, HbC from the other parent. It accounts for almost 25% of all sickle cell disease patients. This type of sickle cell disease will be mild to moderate and anemia may not be as severe as the HbSS type.
Hemoglobin HbS + beta Thalassemia:
Sickle cell disease patients of this type inherit one HbS gene from one parent and another abnormal hemoglobin gene beta Thallessemia from the other parent. It is a mild form of sickle cell disease.
There are other forms of sickle cell disease too, they may carry the same symptoms as that of HbSS-type sickle cell disease but the symptoms may be less severe.
Guidelines for Sickle Cell Children Parents
Dear parents of sickle cell children, please understand that your child has inherited the blood disorder from the faulty Hb genes of both of you. If one parent is a carrier of the sickle cell trait and the other partner is free, the child will not have sickle cell disease. It is only when both parents are carriers of the sickle cell trait, the chances of passing on the faulty Hb genes to your offspring are high. Sickle cell children are born to parents who are carriers of sickle cell trait. Sickle cell disease is not a communicable disease. A child with sickle cell disease may fail to thrive and may have serious complications due to the SCD. The parents of SCD children must be aware of the complications their child may have and must be proactive in avoiding serious life-threatening complications.
Sickle cell disease may have serious complications:
Sickle cell disease is usually managed by providing symptomatic relief from symptoms through frequent blood transfusions, medications (Hydroxyurea, folic acid, pain killers), and occasionally surgeries for life-threatening emergencies. The complications of sickle cell disease can be really serious and children suffering from the severe form of sickle cell disease may fail to thrive or live a normal healthy life. Their development may be seriously compromised. The serious Sickle cell disease complications are:
Vaso inclusive crises (Pain crises):
Pain crises (Vaso Inclusive crises) are a common complication of sickle cell disease. An excruciating sudden sharp and stabbing pain can occur in any part of the body in a sickle cell disease patient. The pain usually affects the chest, back, legs, and arms. This pain may last for a few minutes and up to a few hours. The pain usually comes and goes but if it lasts for more than six months, it is called chronic sickle cell pain.
Acute chest syndrome:
Acute chest syndrome in sickle cell disease patients may occur when the sickled red blood cells block blood flow to the lungs and the oxygen supply is disrupted to lung tissues. It can cause lung injury, difficulty in breathing, and disrupted oxygen supply to the rest of the body. Acute chest syndrome can be a life-threatening medical condition and may require immediate medical intervention.
A stroke:
A brain stroke is a serious complication of sickle cell disease. When the sickle cells clump together in the capillaries supplying blood to brain tissues, they block the oxygen supply to brain tissues. The disruption of oxygen supply to brain tissues will cause a stroke. Stroke is a common problem with sickle cell disease patients. Approximately 11% of people with sickle cell anemia may have strokes by age 20, and 24% may have strokes by age 45. A stroke can cause an irreversible neurological disorder and may hamper the learning abilities of a child.
Splenic sequestration:
In sickle cell disease patients, sickle cells clump together and block the splenic blood vessels. It causes a sudden, painful enlargement of the spleen. Some sickle cell patients will have complete damage to the spleen thereby making it non-functional (autosplenectomy). In most sickle cell disease patients spleen may have to be removed (splenectomy) due to complications of sickle cell disease. Patients without a spleen are at higher risk for infections from bacteria such as Streptococcus, Haemophilus, and Salmonella species.
Multiple organ damage:
Sickle cell disease patients may have multiple organ damage due to sickle cell clumping together and blocking or reducing blood supplies and oxygen to those organs. Vital organs like the heart, lungs, and kidneys can be damaged over sometime in sickle cell disease patients. Sickle cell disease can cause multiple organ failure.
Frequent Infections:
Children with sickle cell disease are prone to frequent infections, such as the flu, meningitis, and pneumonia. Pneumonia is a leading cause of death in infants and young children with SCD. Children with SCD require regular blood transfusions as part of their treatment and are also at increased risk of viral hepatitis.
Vision Problems:
Sickle cell disease patients may have vision problems too. The clumping together of sickle cells may disrupt the blood supply to eye vessels. The blockage affects the retina and may cause sudden loss of vision, if untreated, it can cause a permanent loss of vision.
Priapism:
It is an unexpected and prolonged erection affecting sickle cell disease men. It will affect the penile tissues and blood vessels and may cause permanent damage. A prolonged penile erection that may last more than 4 hours will need medical intervention.
Anemia:
HbSS type of sickle cell disease may cause severe anemia in the patient and will require acute blood transfusions as well as RBC infusion. Sickle cells do not live the full life of the red blood cells (120 days) and may die in 10-20 days thereby causing low RBCs. The bone marrow, which produces RBCc can not cope with the increased demand, and subsequently patient will have severe anemia.
There are several other complications in sickle cell disease patients. Children with sickle cell disease may have slowed development and tinted growth.
Give your child the freedom from serious complications of sickle cell disease. Gift them a curative sickle cell disease treatment in India with our medical tourism assistance.
India is a preferred destination for curative sickle cell disease treatment
India has become the number 1 destination for curative sickle cell disease specialized medical treatments among the parents and relatives of sickle cell disease children from African countries like Nigeria, Tanzania, Democratic Republic of Congo, Uganda, Kenya, Cameroon, Ghana, Rwanda, Middle Eastern countries like Saudi Arabia, Oman, and the Caribbean islands.
Allogenic bone marrow transplants are established curative Sickle cell disease treatments. The low-cost allogenic bone marrow transplants with the highest success rates have propelled India as the number 1 destination for curative sickle cell disease treatments. The reasons for India becoming a preferred destination for curative Sickle cell disease treatment among international patients seeking specialized medical treatments offshore are:
- The lowest cost of allogeneic bone marrow transplant (BMT) among the advanced medical treatment centers in the world
- The Highest success rates for allogeneic bone marrow transplants, among the best bone marrow transplant hospitals in the world
- Well-established and acknowledged competency of the best sickle cell disease curative treatment doctors (Best hematologists and BMT specialist doctors) in India
- The Top sickle cell disease treatment hospitals (best bone marrow transplant hospitals) in India are well-managed with the latest cutting end technologies and state-of-the-art infrastructure for delivering successful allogenic bone marrow transplants for curative treatment of sickle cell disease.
- Availability of flexible rental budget hotels and fully furnished guest houses in a safe and secure environment, close to the top bone marrow transplant hospitals in India
- No waiting period for the commencement of treatment
- Easy and prompt medical visa issuance from the respective consulates and high commissions of India in the patient's country
- Availability of free bilingual interpreters facilities for overcoming the language barrier
- Empanelment of all major international health insurance companies in the top bone marrow transplant hospitals in India
As the best medical tourism assistance company, CMCS Health provides successful curative sickle cell disease treatments in India at the top sickle cell disease treatment hospitals with the best sickle cell disease treatment doctors for foreign patients.
Specialized curative Sickle Cell Disease Treatments:
Children with sickle cell disease may fail to thrive and may not be able to live a full, normal life. Children suffering from HbSS or “Zero” (HbS beta 0), where the sickle cell disease is severe can be provided curative sickle cell disease treatment. Currently, there are two established and approved curative treatments for sickle cell disease.
Allogeneic Bone marrow transplant treatment:
Allogeneic bone marrow transplant has been approved and is an established curative treatment for sickle cell disease patients. An Allogeneic bone marrow transplant is a specialized medical treatment that requires replacing the damaged bone marrow responsible for producing sickle red blood cells with healthy bone marrow from a donor so that the bone marrow of the patient starts producing normal and healthy red blood cells. An allogeneic bone marrow transplant is done with an HLA-matching marrow donor. To Know more about low-cost bone marrow transplant in India as curative sickle cell disease treatment, click on the link below 👇
Curative sickle cell disease treatment - Allogeneic bone marrow transplant. |
As Global Healthcare Guardians for international patients seeking specialized medical treatments offshore, CMCS Health - a leading medical tourism assistance company facilitates and coordinates low-cost allogeneic bone marrow transplants for sickle cell disease curative treatment. Our associated expert and best sickle cell disease treatment doctors are performing successful allogeneic bone marrow transplants routinely to cure sickle cell disease. We have coordinated successfully scores of allogeneic bone marrow transplants for patients from several African countries to treat sickle cell disease. The top sickle cell disease treatment hospitals in India are equipped with the latest cutting-edge technologies and state of art infrastructure to offer the highest success rates in sickle cell disease treatment.
Advantages of Allogeneic BMT as curative sickle cell disease treatment:
Allogeneic bone marrow transplants are well-established and universally acknowledged curative medical treatment for sickle cell disease. Allogeneic BMTs are routinely being performed worldwide for the cure of sickle cell disease. Allogeneic BMT for sickle cell disease curative treatments have distinct advantages:
- A definite cure for sickle cell disease
- Allogeneic BMT frees the sickle cell disease child from acute and chronic complications
- Allogeneic BMT allows a sickle cell disease child to live a normal and full life
- 90% of the sickle cell disease children will get cured of sickle cell disease post a successful allogeneic BMT
- Improves quality of life and overall survival
Limitations of Allogeneic bone marrow transplants:
Although allogeneic BMT is the standard curative treatment for sickle cell disease children it still has certain limitations:
- Finding a suitable HLA matching donor
- The child has to be on immunosuppressive medications to avoid rejection of the bone marrow graft.
- The high-intensity conditioning regimens may cause infertility and secondary malignancies in some patients (although they can be managed with counseling and adopting preemptive measures for overcoming fertility issues and periodic screening to handle any secondary malignancy)
- Risk of GVHD even with a 100% HLA matching sibling donor ( around 9%) and higher with other donors
- The risk of graft rejection ( although the risk can be curtailed by screening the donor-specific antibodies before the BMT)
Careful screening before the allogeneic bone marrow transplant as curative treatment for sickle cell disease patients, selecting the best matching donor, and regular monitoring may help in overcoming these limitations. Proper counseling of parents of sickle cell disease children about the benefits and potential dangers may help them make an empowered decision.
Success Rate for curative sickle cell disease treatment in India:
The bone marrow transplant programs in India have highly evolved during the last decade. The state-of-the-art infrastructure, the latest cutting-edge technologies in both the diagnostics and treatment fields, and the unparalleled expertise of the best Indian bone marrow transplant specialist doctors have delivered the highest success rates for sickle cell disease treatment. The overall success rate for sickle cell disease treatment in India also depends on the type of donor, the age of the patient, and how every individual patient responds to treatment.
Important Links for Curative Sickle Cell Anemia Treatment in India
Parents and caretakers of many Sickle Cell Children prefer India for curative Sickle Cell Disease (SCD) treatment for their children. The High success rate and comparatively low cost of curative Sickle Cell Treatment and other logistics have made India the number one choice among foreign nationals seeking Allogeneic BMT for Sickle cell treatment. 👇
Sickle Cell Anemia (SCD) Treatment for Tanzania Children Sickle cell treatment for African Children | Beyond BT, Hydroxyurea and Folic Acid. |
Gene Therapy as Sickle Cell Disease Curative Treatment:
Gene therapy has recently been approved as curative treatment for sickle cell disease. Gene therapy involves either correcting an abnormal hemoglobin gene or inserting a normal hemoglobin gene into a patient's hemopoietic stem cell. Gene therapy is available only at a few centers in the world. In 2023, the US FDA approved two gene therapies as curative treatments for sickle cell disease. These two gene therapies are used only for SCD patients who are 12 years of age or above.
The mechanism of gene therapy for sickle cell disease treatment consists of removing a patient's stem cells, modifying them by correcting an abnormal hemoglobin gene, or inserting a normal hemoglobin gene into a patient's hemopoietic stem cell and reinfusing it into the sickle cell disease patient. The two gene therapies have different mechanisms.
Exagamglogene autotemcel (exa-cel) Gene therapy for SCD treatment:
Exa-cel gene therapy is a cell-based therapy. The Exa-cel gene therapy modifies hematopoietic stem cells (HSCs) via genome editing using CRISPR/Cas9 technology. This is the first therapy using CRISPR/Cas9 technology to be approved by the FDA. Exa-cel gene therapy modifies hematopoietic stem cells (HSCs) via genome editing using CRISPR/Cas9 technology. The modified stem cells then are reinfused into the patient. The reinfused modified stem cells help the patients produce a healthy form of fetal hemoglobin in place of unhealthy adult hemoglobin. Some researchers feel that CRISPR-Cas9 gene therapy products are better as they’re highly targeted and don’t contain any foreign DNA.
lovotibeglogene autotemcel (lovo-cel) Gene therapy for SCD treatment:
Lovo-cel is the other cell-based gene therapy that uses a lentiviral vector for genetic modification. With this treatment, HSCs are modified to produce HbA, which works similarly to hemoglobin A. Lovo-cel reduces the risk of sickling and occluded blood flow.
Both gene therapies require the patients to undergo myeloablative regimens (chemotherapy) before the modified stem cells are reinfused in the patient's bone marrow.
Advantages of Gene Therapy for sickle cell disease treatment:
- Gene therapy for sickle cell disease treatment is a boon for sickle cell patients who do not have a suitable HLA-matching donor.
- Even elderly sickle cell disease patients can be safely treated with gene therapy
- As it is an autologous type of bone marrow transplant (the patient's bone marrow cells are used for Hb gene modification and reinfused to the patient), the risk of GVHD and graft rejection is avoided with gene therapy.
Limitations of Gene therapy:
Although gene therapy holds great promise for sickle cell disease patients, it has its limitations:
- It is available at very few selected centers and the process is time-consuming ( Patients need to visit the treatment center for at least eight months to one year for completion of therapy)
- The exorbitant cost (1 to 3 million USD) makes it unaffordable for the majority of sickle cell disease patient
- As it is recently approved treatment for sickle cell treatment, the long-term efficacy and side effects are still to be documented
Sickle Cell Disease Treatment Cost in India:
Allogenic BMT cost for Sickle cell disease treatment in India depends on the type of donor, hospital, and the BMT specialist doctors. The average cost of allogeneic BMT for sickle cell disease treatment is as follows:
Initial investigations for 100% HLA matching sibling donor | Around 1000 - 1500 USD |
Cost of allogeneic BMT for SCD treatment with 100% matching sibling donor | 25000 - 28000 USD |
Cost of allogeneic BMT for SCD treatment with Haploidentical ( Half matched - Donor) | 30000 -35000 USD |
Cost of Allogeneic BMT for SCD treatment with MUD (matching unrelated donor) | 30000 - 80000 USD |
The investigation cost post-successful BMT | 500 - 1000 USD/month for 1-2 months. |
Please Note:
- The above cost is as per general estimation, it may increase if any additional treatment is needed
- Cost may increase if any additional conditioning regimen medication is used
- Cost includes only 21 days of BMT isolation ICU stay, cost may increase in case of overstaying in the hospital
- The cost of allogeneic BMT with a matching unrelated donor (MUD) may depend on the country of registry of the donor
Cost of gene therapy for SCD treatment:
The Gene therapy cost at present could be anywhere between 1 to 3 million USD. Moreover, as it is available currently at a few selected centers and the treatment requires multiple visits of the patient to treatment centers for 8 to 12 months, the cost of staying near the treatment centers and logistics add to the overall cost of gene therapy.
Special assistance for foreign sickle cell patients
CMCS Health, the leading medical tourism company, assists foreign sickle cell patients and their parents by connecting them with the best sickle cell treatment specialist doctors and top Hospitals offering curative sickle cell anemia treatment.
- CMCS Health provides free consultation and counseling from the top Sickle cell specialist doctors to parents of sickle cell children.
- CMCS Health assists foreign patients in choosing the best bone marrow donor for their child.
- We can collect the samples from the patients and their perspective donors (siblings) for a high-resolution HLA match and bring it to India for testing.
- CMCS Health assists foreign patients in getting medical visas for coming to India for curative sickle cell disease treatment.
- The dedicated expert patient care executives of CMCS Health, coordinate the entire medical treatment process for foreign patients with due diligence.
- Our patient care experts assist foreign patients with other logistics needed for their child's medical treatment and Indian stay during the treatment.
- A regular treatment follow-up of the patient with the treating Indian doctor after patient reaches back their country post a successful sickle cell treatment.
Best Doctors for sickle cell disease treatment in India:
The best sickle cell disease treatment doctors in India are:
- Dr. Vikas Dua, Pediatric BMT specialist, and hemato. oncologist, Gurugram, India.
- Dr. Satya Prakash Yadav Pediatric hematologist and BMT specialist, Gurugram, India.
- Dr. Pawan Kumar Singh, Hematologist and BMT specialist, Gurugram, India.
- Dr. Rahul Bhargava, Hematologist and BMT specialist, Gurugram, India.
- Dr. Dharma Chaudhary, Hematologist and BMT specialist, New Delhi, India.
- Dr. Gaurav Dixit, Hematologist and BMT specialist, Gurugram, India.
- Dr. Shishir Seth, Hematologist and BMT specialist, New Delhi, India.
- Dr. Reyaz Ahmad, Hematologist and BMT specialist, New Delhi, India.
- Dr. Gaurav Dixit, Hematologist and BMT specialist, Gurugram, India.
- Dr. Balkrishna Padate, Hematologist and BMT specialist, Mumbai, India.
Top Hospitals for Sickle Cell Disease Treatment in India:
Top Sickle cell disease treatment hospitals in India are well-managed and equipped with the latest cutting-edge technologies and state-of-the-art infrastructure with dedicated bone marrow transplant units.
- Fortis Memorial Research Institute, Gurugram, India.
- Medanta - The Medicity, Gurugram, India.
- Sanar International Hospital, Gurugram, India.
- Indraprastha Apollo Hospital, New Delhi, India.
- Artemis Hospital, Gurugram, India.
- BLK Max Super Specialty Hospital, New Delhi, India.
- Nanawati Max Super Specialty Hospital, Mumbai, India.
- Max Super Specialty Hospital, New Delhi, India.
Save your child from the complications of sickle cell disease.
Gift them a curative sickle cell disease treatment. Bless them with a disease-free normal life.