Sickle cell disease is a chronic inherited blood disorder that impacts millions of people across the globe particularly in Nigeria where there are numerous individuals who suffer from the disease. It can result in intense pain, anemia, recurring infections, organ damage and additional issues that have a profound impact on quality of life.
One of the major questions that often come to the mind of the Nigerian family members and patients of sickle cell disease is whether a cure exists. Medical research has led to potentially curative treatments such as bone marrow transplantation and gene therapy, although the main approach has been to treat the symptoms and prevent complications.
This guide provides information about the new treatment options for sickle cell disease in Nigeria and abroad including high-tech treatment that is available in India for those who qualify.
Whether there is any cure for sickle cell disease will vary based upon the kind of treatment.
Until now, sickle cell disease has been thought of as a lifetime course of disease since it is a genetic defect that disrupts the way hemoglobin is made. While conventional drugs can treat the symptoms, lower the risk of complications and extend a person's lifespan, they do not address the underlying genetic cause.
But some advanced therapies may be able to cure the disease in appropriate patients.
The two most important possible curative methods are:
Not all treatments are appropriate for all patients and should be carefully considered and evaluated by the specialist.
Sickle cell disease is a genetic condition of the red blood cells. The red blood cells are normally round and flexible enough to pass through the blood vessels. In sickle cell disease, abnormal hemoglobin makes the red blood cells stiff and sickle shaped.
The abnormal cells can cause blockage of blood flow which can result in:
The disease is passed on from both parents to the child with each parent passing on an abnormal hemoglobin gene.
Treatment of sickle cell disease in Nigeria is largely symptomatic, preventive and quality of life.
The treatment will depend on the patient's age, severity of the disease, medical history and any complications.
Treatment includes the following:
Sickle-shaped cells clog the blood flow to tissues and organs causing pain crises.
Doctors may recommend:
Prompt treatment of pain crises can help minimize complications.
Potential benefits include:
During treatment, regular blood tests and medical monitoring is required.
Some infections are more common in people with sickle cell disease.
Preventive care may involve:
One of the most well-established potentially curative treatments is bone marrow transplantation, however, for many patients the question is, “Is there a cure for sickle cell disease?”
A bone marrow transplant or a "blood stem cell transplant" (HSCT) involves the patient replacing his or her abnormal blood-forming stem cells with normal stem cells from a compatible donor.
These new stem cells can generate healthy red blood cells rather than sickle-shaped blood cells.
The treatment usually has multiple stages:
The doctors review the patient's general condition, the severity of the disease, organ function and the patient's suitability for transplant.
Human leukocyte antigen (HLA) typing identifies an appropriate donor.
When a fully matched sibling donor is available, a fully matched sibling donor is preferred but other donor options may be available.
The patient is given chemotherapy or other “conditioning” treatments to make the bone marrow ready for new healthy donor cells.
They are delivered by means of an intravenous line into the patient's circulation and contain healthy stem cells.
New blood cells start to be made by the transplanted cells. Patients need to be monitored for infections, graft versus host disease and other issues.
For those who have severe sickle cell disease, treatment with bone marrow transplant might be discussed with them when they have some of the following symptoms:
In some cases, children and younger adults may fare better particularly if transplants are done before the organs are severely damaged.
Eligibility is not dependent upon age, though. Each patient should be evaluated individually.
Gene therapy is one of the most exciting advances in the treatment of sickle cell disease.
Gene therapy is different from traditional drugs; the goal of gene therapy is to correct the genetic defect that leads to sickle cell disease.
Medical research is ongoing to find a way to change the blood-forming stem cells of a patient's blood to make healthier hemoglobin.
In general, the process is as follows:
Some gene-editing tactics can boost the production of fetal hemoglobin which can help diminish the effects of abnormal sickle hemoglobin.
Gene therapy is looked upon as a great breakthrough as it could make a matched donor redundant.
However, availability, cost, eligibility and long term follow-up is important.
India is emerging as a key country in the world for patients to come for advanced services in hematology and bone marrow transplantation.
For the Nigerians who want to go for treatment of sickle cell disease in India, they can avail access to specialized hospitals that provide:
Major hospitals in India have expertise in dealing with complex blood disorders like sickle cell disease and other inherited hemoglobin disorders.
Some of the reasons why Nigerian patients opt for treatment in India:
Indian hospitals boast hematologists and bone marrow transplant specialists to deal with complex cases.
Patients may have detailed tests which determine the severity of the disease and eligibility to treatment.
Carefully selected patients may have a long-term option of bone marrow transplantation.
These can range from medical visa support to coordinating accommodation to providing interpreters and treatment planning.
Treatment costs for sickle cell disease are widely variable with the cost differing by type of treatment.
Conventional treatments using medication and regular monitoring might be more cost-effective but can be long-term management.
The cost of bone marrow transplant is higher because of the following:
Special laboratory technology and manufacturing processes make gene therapy much more expensive.
Other expenses may also apply for Nigerian patients who are considering treatment in foreign countries and include travel, accommodation, visa and follow up costs.
The cost of medicine varies from patient to patient and treatment to treatment, so a medical estimate that's personalized to your needs is important.
Recovery following a bone marrow transplant may be a lengthy procedure that lasts for several months.
In the early recovery phase, patients can experience:
Patients might require staying at the transplant centre for the duration of a few weeks or months depending on how they are recovering.
Follow-up is very important to assess blood counts, organ function, immune recovery and complications.
With the progress of medical science, the question of whether a cure for sickle cell disease is possible has been raised even more.
Selected patients have potentially curative options such as bone marrow transplantation and gene therapy. But these treatments cannot be guaranteed to be suitable for everyone.
Traditional therapies are still important for many people and are crucial to help alleviate symptoms and minimise complications.
Treatment will vary according to:
It is important that patients not discontinue medications or to consider transplantation without the advice of a qualified hematologist.
When asked by a family: Is there a cure for sickle cell disease? The answer today is more hopeful than ever. Treatment of sickle cell disease is geared towards the management of symptoms and prevention of complications, although potentially curative therapies such as bone marrow transplantation and gene therapy may be available for eligible individuals. For severely affected sickle cell patients in Nigeria, advanced treatment is possible with specialist consultations in India where experienced hematologists and transplant centers offer in-depth care. Early Assessment and Medical Advice are essential to help identify the best and safest treatment course.
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