Stem Cell Therapy Treatments: Stem Cell Transplant

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Showing posts with label Stem Cell Transplant. Show all posts
Showing posts with label Stem Cell Transplant. Show all posts

May 01, 2018

Stem Cell Therapy Reviving Hopes For Arthritis Patients By Producing Cartilage

May 01, 2018 0

Stem Cell Therapy Reviving Hopes For Arthritis Patients By Producing Cartilage

Adult stem cells have been in use for different biomedical researchers and novel therapeutic applications for a range of diseases since long. Once again, they prove to be a useful medical tool for debilitating conditions as Osteoarthritis. Since researchers have created balanced joint cartilage using adult stem cells, we can expect to get rid of the excruciating joint pain very soon with an effective transplant process. The researchers at the University and University Hospital of Basel created the liquid component known as cartilage applying certain molecular techniques. The process of the tissue development took place during embryonic cartilage formation. Every one of us could have an elaborate view on this report from the scientific journal of PNAS.
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What Osteoarthritis Is And How Does It Affect Us?
Stem Cell Therapy Arthritis

As we all know, osteoarthritis is a degenerative condition of the joints, which causes the destruction of the natural cartilage, weakening of bone density and inflammation. As per medical reports, maximum cases of osteoarthritis result in joint pain restricting the ability to perform normal activities, while affecting the normal quality of life. Until today, so many techniques and medical interventions have proved ineffective in healing the articular cartilage.
In addition to this, mesenchymal stem cell-based therapies have been showing promising hopes to advance cartilage repair. Despite some progressive advancement in the pre-clinical trials, these cell-based therapies are yet to get approval for use in clinical settings. Along with this, other new techniques are on the way to offer novel therapeutic uses for this condition. MSC-based extracellular compounds, improved CRISPR CAS 9 gene editing tools, and miRNAs are devised for the development of new strategies for OA.
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While these are under process, we have another new entrant as a form of stem cell-based cartilage to alleviate our severities.

The Hypothesis Behind The Cartilage Formation

The skeletal tissue regeneration requires specific mesenchymal or stromal cells from the bone marrow-derived adult stem cells. The adult stem cells pass through a transformation stage, where they first become cartilage, and bone tissues later. It is a natural and an automatic process of the stem cells to turn into cartilage, and then enter the ‘hypertrophic’ state. In this specific state, it spontaneously becomes bone tissues. The same state follows during the cartilaginous tissues after a fracture.

How It Works

The research team at The University of and University Hospital of Basel showed that cartilage formation is possible using mesenchymal stem cells. However, the process must adhere to the embryonic development of articular cartilage under the influence of certain molecular processes. In order to achieve the cartilage formation, the technique must restrict the signaling pathway of a certain protein Bone Morphogenetic Protein or BMP.


Further, the scientists highlighted the influence of two highly BMP receptor inhibitors in the whole process. When cultured in a microfluid platform, it is possible for them to form cartilage by blocking the effects of the inhibitors. In addition to this, with a blockage for a short span of time, the cartilage can be developed both in a mouse and the lab models.

Cells Based Models

This discovery is a good sign in the way of disease modeling. At the same time, it opens up new and advanced perspectives for the regenerative medicines and therapies for cartilage formation. Thereby, it focuses on the methods to manipulate tissue engineering for the development of any organ tissue during embryonic cartilage formation.

Last of all, using a certain molecular process, we can control the movement of progenitor and adult stem cells.

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April 23, 2018

Stem Cells: Treating Heart Disease With Umbilical Cord Blood

April 23, 2018 2

Treating Heart Disease With Umbilical Cord Blood

So far, we have enough evidence that the stem cells derived from the umbilical cord blood have the potential to do the magic. The magic is nothing, but in the form of regenerative medicines and therapies, that could help us combat a range of chronic diseases. Now, the researchers at the American Heart Association has once again brought us good news for the patients with cardiovascular issues. The insight of their research has been published in the medical journal Circulation Research.


Until now, we have to use invasive procedures to cure the patients with heart failure complications. But, the latest findings focus to aid in the development of noninvasive therapies using the umbilical cord. By using the therapy, it will be possible to make improvements in the heart muscles and make changes to the quality of the life.

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The  Concern Over The Disorder
heart failure stem cells

When the heart muscle cannot have the ability to pump blood effectively, it is known as heart failure. Nearly 37 million people globally are suffering from this heart disease. We have too many medical advances, still, we cannot treat people with heart complications. And a huge number of patients die every year despite being diagnosed with heart failure symptoms. So, the finding of the study is useful for us to get rid of the disorder.

The Elaborate Finding Of The Research

The trial showed a huge potential in favor of the patients taking part in the clinical trials. As many as 30 patients with lower to moderate level of heart failure complications participate. They aged between 18 to 75. Before these patients could receive intravenous infusions of umbilical cord-derived stem cells, they all had minimal drug therapy. The donors for the trials had full-term developed placentas from which the umbilical cord has been derived. Some of the patients received placebo also to determine the efficacy of the stem cells.
stem cell heart transplant

Consequences Of The Stem Cell Therapy As Compared To The Placebo

  • The patients had an improved life with a flexible capacity to pump blood to the heart and back to the whole body.
  • The quality of life has been
  • There was no side-effect, which is a quite common post any stem cell transplants. This apart, no sign of alloantibodies, or what we know by immune complexes was not detected so far.
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Umbilical cord stem cells did not show any potential though, the current studies are quite promising. We could no longer worry about the adverse effects of the cardiovascular complications, from now onwards. As is with intravenous umbilical cord stem cells, they are easily available, and accessible. Therefore, they raise more positive hopes for us. They are free of all incumbrances as we generally find in case with embryonic stem cells. Since they do not cause any immune complexes, they are a great scientific tool for the scientists for medical advances.

Why Choosing Umbilical Cord In Place Of Drug Therapy?

Such discovery is a way of living life better for many patients with cardiovascular symptoms. Earlier we have many instances which showcase that drug-based therapy is ineffective in case of heart diseases. They are not able to offer maximum benefit to alleviate the heart failure conditions. And that’s being one of the leading causes of patients’ concer, they opt for different invasive therapies. There are heart transplantations and ventricular mechanical assist device therapies, which often prove to be quite pricey for the patients.  

“The finding of the study is quite promising for us, as they could pave the way to develop non-invasive and promising new therapy for the patients with cardiovascular complications,” said the study correspondent author Fernando Figueroa.

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April 20, 2018

Stem Cell Summit:Ways To Improve Anti-CD19 CAR-T Cell Performances

April 20, 2018 0

Why Should We Look Beyond Anti-CD 19 CAR-T Cells? 

In the recent stem cell summit, we had so many strategies to improve the quality of stem cell transplants and make them successful. One of such strategies undertaken in the January Summit, held in Florida raises a concern over the performance of CD19 and finds pathways to improve its efficacy to target solid tumors.


The success of clinical studies on the efficacy of modified T cells garnered our hopes in the development of novel therapeutic treatments. We have witnessed that modification of chimeric antigen receptors (CARs) have been able to defy the cytotoxicity of tumors in many clinical studies. Engineered CD19 CAR-T cells being active in inducing anti-inflammatory immunity against B-cells malignancies, these cells are participating in the development of cancer adoptive immunotherapy.
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However, the hurdle in its way seems to be its ineffectiveness to combat solid tumors. We can find several factors that work against various approaches of these modified T-cells. The first one being the neoplasm activity of the microenvironment, which does not allow CAR-T cells to survive and reach the tumor surface. The host’s defensive mechanism becomes ineffective due to the useless anergic status developing from the ambiguous interactive communication amongst tumor components, stromal and immune cells.


Amidst these confusions, what pathways we should take to improve the performance of CD19 CAR-T cells? Or Should we look beyond some other immunotherapy to treat solid tumors?
stem cell research car-t cells

Efficacy Of CD19 CAR-T Cells

Anti-tumor efficacy of anti CD19 CAR-T cells has been expressed in children and adults through many clinical studies. T-cells can express its anti-immunotherapy properties against a series of B-cell acute lymphoblastic leukemia and other B-cell malignancies. We have two anti-cancer immunotherapies infused with anti-CD19 CAR-T cells named YESCARTA and KYMRIAH. Both these drugs are useful in suppressing B-cell expressions.


While this antigen CD19 CAR-T cell is capable of treating many B-cell malignancies, it is ineffective to even target solid tumor cells. Why so?

Factors Behind CD19 CAR-T Cells Being Inefficient In Solid Tumor Control

Its poor efficacy against malignant epithelial cells is due to the lack of capacity to target specific antigens in the microenvironment. Its inability to target active splice variant of epidermal growth factor receptor (EGFR), causes the growth of solid tumors and creates a hostile nature for its survival.

Ways To Improve Antigen Anti-CD19 CAR-T Cell Performances

While this immunotherapy fails to provide a positive result for the patients with solid tumors, other antigens like anti GD-2 CAR-T cells have shown some promises to treat different malignant conditions in clinical trials. Therefore, we can demand more clinical trials which can bring forth the introduction of anti-cancer immunotherapy using antigens.
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By using IL-2 support and increasing the number of cells for the ACT in melanoma, we can achieve a positive result for patients with solid tumors. Simultaneously, the physical barriers created by epithelial and mesenchymal cells need to be restricted to prevent ill effects of the cytotoxic functions of the solid tumors. And to effectively increase its efficacy against solid tumors, its persistence, eruption in the environment and tumor homing should be increased.

At the same time, if this antigen underperforms despite some changes, we should look beyond this. A recent study showed that BCMA targeted CAR-T cells can effectively perform on tumor-specific cells to stop further spread of the diseases. Many clinical trials have shown promises that this antigen could provide adequate relief from multiple myeloma.

So, we can rest assured that clinical studies and research could bring us effective CAR-T cells based immunotherapy in order to combat multiple malignancies and myeloma in future.

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April 12, 2018

Mesenchymal Stem Cell Therapy to Increase the Survival Rate in HLHS

April 12, 2018 0

Mesenchymal Stem Cell Therapy to Increase the Survival Rate in HLHS

Mesenchymal stem cell therapy seems an appropriate answer to the severities of HLHS disease. The congenital heart defects hypoplastic left heart syndrome (HLHS) does not allow babies to survive more than an average of five years after the birth. For the heart surgeons, it is one of the most challenging and complex forms of heart disorder to treat using surgeries and heart transplant.

Hypoplastic Left Heart Syndrome

The disorder leaves us with an incomplete developed left ventricle in the heart, which takes a key role in pumping blood to the lungs. As per the Centers for Disease Control and Prevention, every year 960 babies are exposed to this disorder, and if left untreated the outcome is always fatal.
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In normal, when we have healthy hearts, the right ventricle does not have to work hard to supply deoxygenated blood to the lungs from the body. The left ventricle, on the other hand, uses a greater level of strength to pump resupplied blood back to the body. However, with HLHS, the right side of the heart has to take the double workloads, as well as maintaining the blood circulation to both lungs and body. So, when the stress is too much for the right side of the heart, the heart fails to work and causes the baby to die.

Type of Treatments

HLHS requires three types of surgeries in order to be treated completely- the first one performed at the birth,  the second one at 4 months of age and the final one at the age of 2 or 4. However, it does not always promise to offer a curative treatment as patients with this disorder are more likely to die even after a heart transplant.
stem cell transplant for babies

Since open heart surgeries or transplant are not providing promising results, the only curative treatment option appears to be the stem cell therapy


We have more instances that support the use of stem cell therapy in the treatment of HLHS in babies. In a more recent incident, surgeons in Maryland injected the cells derived from bone marrow of the donor into the heart of baby Autumn Brown with HLHS. The four-month baby Autumn is now a part of the stem cell study with 10 babies at the  University of Maryland Medical Center. Both Wayne Brown and LeeAnn Janes, Autumn’s parents are hopeful for a better outcome of the clinical trial for their baby. As Autumn ’s mother wants nothing more than her healthy life, she expects that she will lead a normal life with implanted stem cells in her heart. More instances show that she is improving and will meet her relatives soon. Janes said, “I could see the stem cells as creating a super heart.”
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The Finding of Their Study

The research carried out by Dr. Kaushal had an objective of boosting or regenerating the right ventricle in patients with HLHS so that their right heart could pump blood strongly. Their research also showed that babies with implanted mesenchymal stem cells are doing well.
hlhs stem cell transplant babies

MSCs have allogenic properties, so they can easily expand in the culture, and are a perfect source for the trail. MSCs prove to be effective in restoring heart functions in a clinical trial, which focuses the same features for HLHS too. These cells are capable of forming heart muscles and restraining the heart muscle from dying.


The finding of the study suggested that the same techniques will work for patients with hypoplastic left heart syndrome too.

We have earlier seen that stem cells provide a great potential to regenerate heart muscles. So it is believed that the result of the study will work better in case of pediatric patients too. That’s because hearts in younger individuals have better opportunity to grow as opposed to the older patients since the body is still in the development phase.

So, we can hope this novel therapy will one day improve the health of little patients who have a vulnerability to mortality.

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March 23, 2018

Stem Cell Transplant:Treating Bare Lymphocyte Syndrome

March 23, 2018 0

Treating The Conditions Of Bare Lymphocyte Syndrome With Stem Cell Transplant

Bare Lymphocyte Syndrome or most popularly known as MHC Class II deficiency is a rare form of immunodeficiency disease. Children are at high risk as the disorder leaves its impact mostly on them. The disease may attack the patients when there is either deficiency of HLA Class I or Class II antigens on the cell surface.The allogeneic stem cell transplant is the only curative treatment options for this immunodeficiency disorder.

Health Complications Of The MHC Class II Or BLS

The occurrence of the disease may defer your body from developing responsible genes through chromosome 6. It hinders antibody from forming and restricts the cells from responding to the specific antigens. It is evident to experience more physical complications in the respiratory and digestive tract. And as a result, your normal life finds it difficulty in breathing and digesting food.
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More severe complications are there for you when you develop the immunodeficiency disease. You are likely to develop hypogammaglobulinemia, which results in recurrent viral infections caused by parasites and bacteria.

Treatment Choices for BLS

Utero Stem Cell Transplant
You could find a treatment solution for this disease from allogeneic or HLA matched stem cell transplant. However, the survival rate of the patients is highly dependent on the donor cell types.  HLA matched donor transplant improves the survival quality, while the unmatched donor cell transplants offer you 50% of survival success.

Hindrances to SCT for BLS

The relief from the debilitating complications of the disease finds its way through a successful allogeneic stem cell transplant. On failure of the transplant, most children may succumb to infections in their first phase of life.
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Other than this, the alternative donor transplant does not suit the process of the Stem cell transplant. Similarly, other types of immunodeficiency diseases do not benefit from this process as well. Till date, the stem cell transplant using alternative donors increases the chances of Graft-versus-host-disease reactions, incessant graft rejections, and even mortality due to infections.

Study Favoring The SCT For Curing Bare Lymphocyte Syndrome

If you look at the finding of a study carried out on the 16 diseased children, you will have a clear idea about the efficacy of the SCT.

In 1990, 16 affected children were ready for the transplant with the samples. The specimens were derived from HLA unrelated family member, HLA matched adult donor and mismatched cord blood donor. These patients were under treatment in four different medical centers.
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None of the patients had experienced chronic GVHD after the first transplant. However, five patients who underwent the second phase of transplant experienced graft failure. And six patients amongst them developed acute GVHD complications. In spite of that, Stem cell transplant seems to offer the best survival results at 69% of success rate for the children.


Another process for the treatment of bare lymphocyte syndrome is utero stem cell transplant. Many fetal disorders or immunodeficiency disorders like BLS gain good benefits from the utero stem cell transplant.

Utero Stem Cell Transplant

The possible treatment for BLS depends on the maternal T-Cell to be injected intravascularly. With the use of the maternal cells, the results achieve the highest chimerism levels. The robustness of the transplant remains stable for a period of two years with no traces of GVHD. This apart, the utero stem cell transplantation offers many curative treatments for dreadful diseases.

This therapy also reduces the relapse complications caused due to GVHD. Simultaneously, the donor cells receive a better environment to develop in the host fetal cells.

So, we can expect more of such therapies to relieve the health compilations of BLS at the earliest soon after the transplant takes its course.

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March 07, 2018

Type 1 Diabetes:Regenerative Stem Cells To Regrow Beta Cells

March 07, 2018 1

Regenerative Stem Cell Therapy To Regrow Beta Cells For Type 1 Diabetes

Thousands of progenitor cells inhabit the human pancreas. And they can breathe new life into many glucose-responsive beta cells. A cultural process at the laboratory can aid in the whole process. The scientists at the Diabetes Research Institute are seeing some possibility in this new finding. The development of glucose-responsive beta cells using progenitor cells is useful in developing regenerative cell therapies. And the patients with type 1 diabetes can receive a permanent and biological cure for this type of regenerative stem cell therapies.
Type 1 Diabetes

Why Is Type 1 Diabetes A Concern?


Diabetes is one of the widespread and daunting glucose disorders. The prevalence of the glucose immune disorder is rapidly increasing around the world. This is a condition, which is undying as long as the patient survives. Its dominance is high amongst the children. Type 1 Diabetes means living with a regular administration of insulin, followed by long-term of health issues.

It is an autoimmune disorder, which occurs when there is a vast destruction of the pancreatic β cells.

This is how therapies and new treatment option advocates for the restoration of the remaining beta cells, and regulation of T cell autoimmunity.
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Treatments for Type 1 Diabetes


Patients diagnosed with Type 1 Diabetes have to rely on exogenous insulin. An extreme need for insulin becomes obvious when the immune system damages the insulin-producing cells of the pancreas.   However, when the condition deteriorates like glucose level rises above the normal, it seeks the intervention of medication rather than insulin.
diabetes insulin

Islet transplantation comes to the rescue of the patients during the situation. The surgery of islet transplantation is very popular, which needs the donor’s islet cells or the whole pancreas. It is able to regulate the blood glucose level, and no more insulin injections are required. But, there are scarcities of enough donor cells to treat the conditions for the patients with type 1 diabetes.

Stem Cell Transplant For The Cure Of Type 1 Diabetes


The islet transplant may cause many side effects and also lead to the failure of the transplant surgery.  Researchers have highlighted the other source of fruitful treatments. And it lies on the stem cell transplant. By developing more pancreatic cells from various sources like embryonic cells, pluripotent and adult stem cells, the curative treatment options are possible for type 1 diabetes. And the most effective options seem to lie in the regeneration of the insulin-producing cells from the patients themselves. Through regenerative therapy using the patient’s own pancreatic cells, it will eliminate the need of transplanting donor tissues. It will also be able to remove other hurdles from the treatment pathway.

How The Progenitor Cells Work To Develop Regenerative Therapy

Progenitor cells are capable of renewing islets in the pancreas. At the same time, they are proliferative, and hence they can turn them into beta-glucose cells. The ability to renew beta cells in the pancreas, it can lead to the development of the therapeutic treatment for the patients with type 1 diabetes.

The bone morphogenetic protein – 7 or (BMP-7) maintains the growth of the cells, and responsible for stimulating the progenitor-like cells. The cells in pancreas also respond to this protein. Other than this protein, PDX-1 and ALK3 involve in the development and regeneration of the beta cells. Therefore, it is possible to grow proliferative pancreatic cells in the presence of BMP-7 using ‘molecular fishing’ in the native pancreas. And with PDX 1 and ALK 3, they help them differentiate into beta cells.

With the regenerative medicine therapies, the native pancreas is able to restore insulin production. It eliminates the chances of transplant of insulin-producing cells and also the transplant of the pancreas.

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February 20, 2018

Hematopoietic Stem Cell Transplant For Aplastic Anaemia

February 20, 2018 1

Hematopoietic Stem Cell Transplant Can Free Patients From Hazards of Aplastic Anaemia

Aplastic Anemia is a disorder of bone marrow failure. The therapeutic relief from this disorder depends on the hematopoietic stem cell transplant and Immunosuppressive therapy.

More on to AA, it is a type of blood disorder, occurring from the bone marrow failure. The hypocellular bone marrow and relentless pancytopenia characterize patients with AA.  The occurrence of this disease attacks one’s immune system. And as a result, the bone marrow cannot produce marrow cells. Further, it also restricts the production of the blood platelets as well as red and white blood cells.


The prevalence of Aplastic Anemia is high amongst the pediatric children. But, it is not averse to the adults also. However, pediatrics can expect better treatment results with stem cell transplants.
stem cell transplant aplastic anaemia
Source- Flickr

Complications of AA

Frequent blood transfusion is common amongst the patients with AA. The process keeps the blood platelet counts in check. Simultaneously, immunosuppressive therapy or IST offers a better relief by curbing the autoimmune responses. And this way, it prevents causing more damage to the blood-producing cells. Patients need to take another drug therapy to avoid infections. They need to prevent contact with the outer world as well to avoid the contamination of the infection. Sometimes, these drugs do not provide any relief, thus patients are more likely to die of infections.

Treatments Options

Immunosuppressive therapy and matched bone marrow transplant offer treatment options. Well, immunosuppressive therapy or drugs are not without any relapse risks. And hence, stem cell transplantation is considered one of the best curative treatment options for the patients with AA. According to reports published online by Biology of Blood and Marrow Transplantation, patients receiving partially matched bone marrow combined with the high doses of chemotherapy are more likely to survive the disease.

Advantages of Hematopoietic Stem Cell Transplants

  • Transplantation reduces the further complications of Aplastic Anemia.
  • It reinstates the production of the blood cells and platelets.
  • The complications of the Graft-Versus-Host-Disease (GVHD) after the transplant are mild.
  • With immunosuppressive therapy, GVHD complaints can see the exit door.
  • It completely reduces the necessity of further medications.
  • Patients can enjoy their normal life as earlier, and participate in their daily activities.
  • Evidence Validating The Efficacy Of The Hematopoietic Stem Cell Transplant For AA
  • Hematopoietic Stem Cell Transplant (HSCT) is the first choice for the clinical experts when immunosuppressive therapy fails to perform.

And hence, stems from a sibling or matched donor are taken into account for the transplants. However, patients with half donor could also go for the transplants. A study between 2011 and 2016 showed the efficacy of the HSCT using a matched donor, half matched and unrelated matched donor.

Four types of patients received cells depending on the availability of the donors respectively. The patients received drug administration to make their body accept the bone marrow. After it, all the four patients underwent transplants with a half-matched donor, matched donors, and unrelated donor.

A few days after, they received chemotherapy. They stopped the immunosuppressive therapy too after taking it for a year.
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Outcomes Of The Study

Further tests on the patients suggested that there was no need for blood transfusion.
The blood cells, including the platelets, came back to normal.
No fresh requirement of medications was noticed after the discontinuation of immunosuppressive therapy.
The rate of GVHD was less than normal as well. Only a few received the after-effects of the stem cell transplants.
The further IST ensured to reduce the GVHD complaints after a few months.

Future Hopes
bone marrow transplant
Source- Flickr

The shortage of matched donors can no longer be a hurdle in the way of transplants. For patients having half-matched donors or unrelated donors, HSCT is possible using chemotherapy drug cyclophosphamide. The drug is effective in destroying the diseased blood cells and producing new blood cells.

The bone marrow transplants are costly, though, they are most effective treatment options using a full or half-matched donor.

They are indeed life-saving, at the same time, they reduce the additional expenses entailing to IST, blood transfusion, and hospitalizations.

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February 04, 2018

Hematopoietic Stem Cell Therapy Transplant At The Rescue Of Acute Myelofibrosis

February 04, 2018 1

Hematopoietic Stem Cell TherapyTransplant At The Rescue Of Acute Myelofibrosis

Stem cell therapy: Hematopoietic cell transplant (HCT) has all the powerful elements in its treatment procedures to offer better and curative treatment opportunities for patients suffering from acute Myelofibrosis. This is a rare kind of cancer, which prevents the growth of blood-producing cells, and destroys the existing and new cells more rapidly. And as a result, the bone marrow develops scars with the development of fibrous tissues within the bone marrow. So, with the occurrence of this disease, a series of health complications occur in the patients.

Health Complications of Acute Myelofibrosis

This type of leukemia belongs to the group of Myeloproliferative Neoplasms. About 30,000 of the USA citizens are suffering from this disease when their blood begins overproducing the red blood cells. So, the cells cannot produce enough blood in the body, and the body tries to move the blood production to the spleen and liver. And as the bone marrow cannot perform well, it pressurizes the spleen and liver and enlarges them. The disease causes blood clots and extreme bleeding to the people with acute myelofibrosis.
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Other complications include weakness, fatigue, anemia, abdominal pain, low platelet counts and prevalence of infections.

People between 50 and 70 are at high risk of developing this disease.

Stem Cell Therapy Treatment Options for Acute Myelofibrosis

The treatment options depend on the symptoms and the intensity of the complications. There are different drugs used to treat this disease.

A drug called Ruxolitinib targets the inhibitors known as JAK2 and other associated mutants. The chemical compounds of the drug effectively work and restrict the functions of the mutant protein tyrosine kinase, which can be transformed and active in people with myelofibrosis.

There are other drugs too, such as danazol, thalidomide, and erythropoietin. The most effective and the curative treatment process seem to be the stem cell therapy transplant or the bone marrow transplant. However, a fewer number of patients can benefit from the transplant, which also involves treatment-related morbidity and mortality.

stem cell transplant
Image- Flickr.com

Varied Stem Cell Therapy Options for the Treatment of Myelofibrosis

Allogeneic hematopoietic cell transplantation (HCT) is the major treatment choice for myelofibrosis. As older patients cannot stand the toxicity of the Full-Intensity Conditioning (FIC) because of its high rate of non-relapse mortality (NRM), healthy and young patients are not advised to opt for this option. This is the reason why Reduced-Intensity- Conditioning (RIC) provides much better treatment options for MF.

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Candidacy for RIC

Since Myelofibrosis is a disease of elderly people, and it triggers more risks associated with poor transplantation results. So, the determination to perform RIC prior to HSCT relies upon several variables such as - patients and disease-related parameters, performance capability, age, and complication biology as per the standard Dynamic International Prognostic Scoring System or (DIPSS). The determination of the candidature for the RIP is based on the risk factors. They are as follows as below:

  1. >65 years age
  2. Hemoglobin count <10g/dL
  3. White blood cell counts >25x109/L
  4. Peripheral blood blasts ≥ 1% and
  5. Constitutional signs
Patients with low or intermediate-1 risks factors may opt for the stem cell therapy transplant as per another scoring system, Dynamic International Prognostic Scoring System, but they are not without any side effects. Certain mutations such as JAK2, MPL, and CALR cause the occurrences of primary myelofibrosis in the patients with low or intermediate-1 risk, cannot opt for the RIC. Contrary to it, patients with inhibitors or mutant proteins such as SRSF2 and ASXL1 require an instant transplant.
bone marrow transplant

Eligibility of the Donor

The type of donor determines the survival rates for the patients. According to various studies, human leukocyte antigen (HLA), matched donor, preferably with siblings is the ideal donor type. It offers 75% of successful results for 2-year overall survival. It is followed by the matched unrelated donor. With this type of donor, the survival rate becomes weaker and 32% success rate is established for 2-year overall survival. And with the RIC, patients with matched donor transplant can survive for five years at a percentage of 56, matched unrelated donor survival rates is at 48%, while 34% of success rate is established for the mismatched unrelated donor type.

It is uncertain if the Splenomegaly offers any survival opportunities, prior to SCT. It leads to several health complications, including impairment of the liver functions.


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I am an avid health Blog writer. I write for several health sites and blogs, do thorough research work and accumulates all essential information that makes the blogs informative and enhances their value.