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Bone Marrow Transplant Clinic / Pediatric

Today, the treatment of many hereditary or non-hereditary diseases is possible with Stem Cell Transplantation.

Stem Cell Transplantation is a treatment method that can be performed by a difficult, experienced and specialized team. Stem cell transplantation, which is the definitive treatment method for many diseases that cannot be cured by medical treatment, can only be performed in Stem Cell Transplantation Units with special physical conditions.

Patients who are candidates for stem cell transplantation receive intensive treatment before and after transplantation to get rid of their diseases. The most vital of the numerous side effects of these treatments is a weakening or even total lack of the immune system. Even a simple infection can be life-threatening in immunocompromised patients. For this reason, stem cell transplant patients can be transplanted in private clinics with filtered air, single isolation rooms, controlled and limited entry and exit, and strict hygiene rules accepted worldwide. Another feature of these clinics is that they are Intensive Care Units.

Childhood transplants are the treatment method for many diseases. The most common of these are:

  • Leukemias
    • Blood Production Disorders
    • Thalassemia (Mediterranean Anemia)
  • Sickle Cell Disease
  • Bone Marrow Failure
  • Genetic Diseases
  • Immune System Diseases
  • Tumors

Yeditepe University Hospital Pediatric Bone Marrow Transplant Clinic was put into service in November 2015 as a unit with the aforementioned qualifications. The head of the center licensed by the Ministry of Health, Prof. Buket Erer Del Castello has worked in stem cell transplant abroad since 1990 and was awarded the Order of Merit by the President of Italy.

What is Hematopoietic Stem Cell / Bone Marrow Transplant in Children?

Stem cells are cells that play a role in the development and regeneration of all tissues and organs in our body throughout our life. Stem cells can multiply, divide, and differentiate. The most important feature that distinguishes stem cells from other cells is that it creates new stem cells like itself with its unlimited division ability. Every system and / or organ in our body has a stem cell. The main cell that gives birth to the shaped elements of blood is called Hematopoietic (Blood Forming) Stem Cell. The production site of Hematopoietic Stem Cells is bone marrow. According to the needs of the body, while forming new stem cells like themselves, they differentiate into precursor cells that will form shaped cells with different functions in our blood such as red blood cells, thrombocytes and lymphocytes.

 

Childhood is a period that includes the first 18 years of life after birth. Unlike adults, hereditary (inherited) diseases in childhood constitute an important group of diseases. In our country where consanguineous marriages are common, the rate of inherited diseases is high. A significant portion of these life-threatening diseases that involve different systems and organs are treated with hematopoietic stem cell transplantation in childhood.

What Are the Sources of Hematopoietic Stem Cells?


Today, there are three sources of hematopoietic stem cells from which healthy cells are obtained.

Bone Marrow Transplantation
If bone marrow is preferred as the cell source, cell collection is performed in the operating room under general or epidural anesthesia. General anesthesia is preferred in pediatric donors.


Cord Blood
It is ready as it is collected during birth and stored under suitable conditions. The number of cells in the cord blood is limited and may be insufficient depending on the body weight of the recipient (patient). Therefore, its use in pediatric patients is more common.


Peripheral Blood (Blood Circulating In The Veins)
The amount of stem cells circulating in peripheral blood is less than bone marrow. For this reason, if peripheral stem cells are preferred as a stem cell source, the patient or donor is given a drug under the skin (with a needle) for 5 days to increase the amount of cells. On the fifth day, the patient or donor is put on a machine. The increased number of stem cells are collected in special bags.

Types of Stem Cell Transplantation

What are the Types of Hematopoietic Stem Cell / Bone Marrow Transplant?


In stem cell transplantation, the patient is defined as a “recipient” and the person who gives healthy stem cells to the patient is called a “donor”. When classified according to donor characteristics, there are 3 types of hematopoietic stem cell transplants.

Autologous Hematopoietic Stem Cell Transplantation
The type of transplant in which the patient’s own cells are given is defined as “Autologous Stem Cell Transplant”. It is frequently used in the treatment of some solid tumors.


Allogeneic Hematopoietic Stem Cell Transplantation
The type of transplantation performed by taking stem cells from another donor is called “Allogeneic Stem Cell Transplant”. It is the preferred type of transplantation in malignant and genetic diseases.


Syngenic Hematopoietic Stem Cell Transplantation
Transplantation between monozygotic (identical egg) twin siblings is called “Syngenic Stem Cell Transplant”.

In stem cell transplantation, the patient is defined as a “recipient” and the person who gives healthy stem cells to the patient is called a “donor”. When classified according to donor characteristics, there are 3 types of hematopoietic stem cell transplants.

 

Autologous Hematopoietic Stem Cell Transplantation

The type of transplant in which the patient’s own cells are given is defined as “Autologous Stem Cell Transplant”. It is frequently used in the treatment of some solid tumors.

 

Allogeneic Hematopoietic Stem Cell Transplantation

The type of transplantation performed by taking stem cells from another donor is called “Allogeneic Stem Cell Transplant”. It is the preferred type of transplantation in malignant and genetic diseases.

 

Syngenic Hematopoietic Stem Cell Transplantation

Transplantation between monozygotic (identical egg) twin siblings is called “Syngenic Stem Cell Transplant”.

Chronic Leukemia and Its Types

Which Diseases Can Be Treated With Hematopoietic Stem Cell / Bone Marrow Transplantation?

A large number of genetic (hereditary) and non-genetic diseases in children can be treated with hematopoietic stem cell transplantation.

Hematological Malignant Diseases

  • Acute Lymphoblastic Leukemia
  • Acute Myeloblastic Leukemia
  • Chronic Myeloid Leukemia
  • Juvenile Myelomonocytic Leukemia
  • Myelodysplastic Syndrome
  • Non Hodgkin Lymphoma
  • Hodking’s lymphoma.

Primary Immune Deficiencies (Hereditary Immune System Deficiency)

  • Severe Combined Immunodeficiency (SCID)
  • Adenosine Deaminase Deficiency
  • ZAP70 deficiency
  • Omenn Syndrome
  • Chediak Higashi Syndrome
  • Wiscott Aldrich Syndrome
  • Di George Syndrome
  • IPEX Syndrome
  • Autoimmune Lymphoproliferative Disease (ALPS)
  • Chronic Granulomatous Disease

Other Inherited (Hereditary) Diseases

  • Thalassaemia major
  • Sickle Cell Anemia.
  • Fanconi Aplastic Anemia
  • Diamond Blackfan Anemia
  • Primary Hemophagocytic Lymphohistiocytosis
  • Congenital Dyserythropoietic Anemia
  • Kostmann Disease

Bone Marrow Insufficiency (Hematopoietic Stem Cell Defect)

  • Acquired Aplastic Anemia
  • Structural Aplastic Anemia
  • Dyskeratosis Congenita
  • Shwachman Diamond Syndrome
  • Amegakaryocytic Thrombocytopenia
  • Paroxysmal Nocturnal Hemoglobinuria

Autoimmune Diseases (Patients Not Responding to Standard Therapy)

  • Crohn’s disease
  • Systemic Lupus Erythematosus (SLE)
  • Rheumatoid Arthritis
  • Evans Syndrome
  • Autoimmune Hemolytic Anemia

Metabolic diseases

  • Wolman Disease
  • Mannocytosis
  • Mucopolysaccharidosis
  • Leukodystrophies
  • Adrenoleukodystrophy (ALD)
  • Metachromatic Leukodystrophy (MLD)
  • Globoid Cell Leukodystrophy (Krabbe)
  • Osteopetrosis (Metabolic Bone Disease)

Lymphoma Types and Treatment

Symptoms, Diagnosis and Treatment of Multiple Myaloma Disease

How a Donor is chosen for Hematopoietic Stem Cell/Bone Marrow Transplant?

One of the most important factors limiting the application of Hematopoietic Stem Cell Transplantation is that the patient is not HLA compatible (tissue type compatible) stem cell donor. The tissue group is compatible in fraternal twins and siblings who have received the same genetic inheritance from the mother and father.

The ideal donor is a tissue-type matching sibling. With the contribution of technology, science and experience, partially compatible intra-family transfers are made to patients who do not have compatible siblings. For patients without family donors; Tissue-type compatible donor screening is performed from donor banks in our country and voluntary donors registered in the World Bone Marrow Bank and banks where voluntary donated umbilical cord blood is stored. When a compatible donor or cord blood is found, the patient is transplanted with stem cells.

How is Hematopoietic Stem Cell / Bone Marrow Transplant Done?

Stem Cell Transplantation is a multidisciplinary treatment method applied for the treatment of life-threatening diseases that do not heal with standard treatment. If the patient is a child, the legal guardian (mostly parents), if the patient is an adult, the patient himself is informed verbally/in writing about why, when and how this treatment will be carried out, the results, early and long-term risks, and the transfer procedures are started with written consent.

After the transplant decision is made, the type of transplant to be applied, the stem cell source to be used and the healthy donor who will give the stem cell are determined. Then the patient and the donor are prepared for transplant.

Patients who receive chemotherapy before transplant and whose immune system is weakened undergo transplantation in a sterile unit, in specially designed rooms with intensive care unit features. In order to minimize the risk of infection in the unit, entrances and exits are limited and visitors are not accepted. In addition, only devices belonging to this unit are used in the service.

What Should The Patient Pay Attention To After Hematopoietic Stem Cell / Bone Marrow Transplantation?

Patients are closely monitored, especially in the first months after hematopoietic stem cell and bone marrow transplantation. Discharged patients and their relatives are informed in detail about the issues that need to be considered after the transplant.

Some of the points that the patient should pay attention to are as follows:

  • Attention should be paid to the risk of infection. For patients with a weakened immune system, a simple infection poses a life-threatening risk. Therefore, it is necessary to be careful about the following issues until the immune system recovers itself (at least 6 months).
  • Attention should be paid to personal hygiene.
  • Mask should be used when going outside.
  • Should not be in crowded environments.
  • Environments and people at risk of infection such as hospitals and shopping malls should be avoided.
  • Cooked food should be consumed.
  • Attention should be paid to hygiene rules such as not consuming food sold out in the open.
  • The diet recommended by the doctor should be followed.
  • In pediatric patients with hematopoietic stem cell transplantation, the vaccination schedule recommended by the doctor must be followed.
  • In long-term follow-up, issues such as growth and development in pediatric patients and school success are important, unlike adults. Families should monitor the child closely and psychosocial evaluations should be done as well as medical check-ups.

How to Apply for Hematopoietic Stem Cell / Bone Marrow Transplant?

Domestic Patients

Anyone who is a citizen of the Republic of Turkey and the need to transplant stem cells, may undergo hematopoietic stem cell transplant under the state guarantee. The patient can be transplanted in the center where he is diagnosed, followed up and treated. If there is no transplant center in that hospital, they are directed to another hematopoietic root cell transplant center recommended by the doctor who treats and follows up or prefers them.

 

Patient Applying from Abroad

The hospital’s overseas patient services team transmits the discharge/examination reports of patients applying from abroad to the doctor in charge of the transplant center. If the doctor evaluating the patient approves, the organization of the patient’s bone marrow transplantation is carried out by the relevant team.

Frequently Asked Questions About Hematopoietic Stem Cell / Bone Marrow Transplant

How Long Does Hematopoietic Stem Cell / Bone Marrow Transplant Take?

Preliminary preparations are made by evaluating the type and stage of the disease before the transplant, and the performance of the recipient. The patient is prepared for transplantation with a chemotherapy and / or radiotherapy protocol called “preparation regime”. These protocols take 8-10 days. Hematopoietic stem cell transplant is not an operation. The hematopoietic stem cell source (bone marrow, peripheral blood or cord blood) planned to be transplanted is given to the patient via vascular access after chemo / radiotherapy. “Nesting” is the spread of these cells through the blood, reaching and settling in the bone marrow. “Bone marrow patching” occurs when donor stem cells, which are placed here, begin to produce healthy blood cells. Although this process takes an average of 2 weeks, it may be shorter or longer. If no complications are seen after the bone marrow patch, the patient is discharged after 4 weeks. In case of complications, the duration of hospital stay may be longer.

Are There Any Harm to Bone Marrow Donation?

Donors are selected from healthy people. However, the donor, which passed all the examinations before transplantation, is evaluated according to the stem cell collection process. Donor is informed about all transactions and processes to be carried out afterwards. For peripheral blood collection, the donor may have bone pain, the features and duration of the collection process are explained by the apheresis doctor at the preparatory stage. All these risks are not at a level that would impose the procedure to save a person’s life. After the bone marrow and peripheral blood stem cell collection procedures, the healthy bone marrow of the donor completes the deficiency of the cells taken from him in a short time.

How Is Bone Marrow Obtained From The Donor?

In patients where bone marrow is preferred as a cell source, cell collection is performed in the operating room under general or epidural anesthesia. In the process of collecting stem cells circulating in peripheral blood, the donor is tied to a machine and the cells are collected. Cord blood is ready as it is collected during birth and stored under appropriate conditions.

 

Who Can Become a Volunteer Donor?

To become a volunteer stem cell donor; It is enough to have a body weight of 50 kg and over between the ages of 18-55 and be healthy.

Relative donor:

  • Sibling donor: The most suitable donor is the HLA matching sibling donor.
  • Relative donor: In societies with frequent consanguineous marriages, HLA matching consanguineous donors can be found.

Unrelated donor (voluntary donor): Compatible donor screening is performed in bone marrow banks where healthy donors are registered in our country and around the world. In addition, suitable cord blood can be found through cord blood banks where healthy cord blood donated voluntarily is stored frozen.

 

What Are the Factors Affecting Results and Success in Stem Cell Transplantation?

Many factors such as the underlying disease and the stage of the disease, the patient’s performance during the transplant, the degree of patient-donor match, the type of transplant, the gender and age of the donor affect the success of the transplant. In general, the disease being at an early stage, the general condition of the patient being good, the patient and donor being young affect the result positively.

Chronic Leukemia and Its Types

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