PHOTO GALLERY THE FIRST BONE MARROW TRANSPLANT

Two months ago, at the Department of Spinal Cord Transplant of the Fundeni Clinical Institute, a spinal cord transplant was successfully performed for a child with beta-thalassemia major, the first of its kind in our country. The beneficiary of the transplant is a 7-year-old girl from Dolj County, addicted to blood transfusions performed monthly from the age of 1 year; received the bone marrow transplant from the family donor - the older sister, aged ten, informed specialists from Fundeni Hospital, on Monday, in a press conference.

Beta-thalassemia major is a serious form of hereditary anemia, which progresses fatally in the second decade of life without proper treatment. It is estimated that there are 350-400 children and adolescents with this disease in Romania.

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Before the transplant, the patient is treated with repeated blood transfusions at average intervals of one month. With the chelating treatment of iron, one of the major risks for the patient is overloading all organs with iron, which is excessively toxic to cells.

Today, the only known definitive treatment for the disease is an allogeneic bone marrow transplant (from a donor).

Transplantation in the patient with beta-thalassemia has some notable features:

Before transplantation, transfusions should be performed with a high degree of compatibility; the sensitization of the recipient (production of antibodies) may lead to the failure (rejection) of the graft;

At the time of its performance, the patient must be in good condition of all body functions, which depends on previous treatment of very good quality of iron chelation;

the transplant is performed in the best conditions with the bone marrow and not with the stem cells harvested from the periphery; this involves general anaesthesia administered to the donor, followed by bone marrow harvesting by repeated bone punctures in the pelvic bones performed in the operating room;

Post-transplant and the prognosis

the evolution of the post-transplant and the prognosis of the patients depends on the degree of damage to the critical organs - and first of all to the liver; survival after transplantation without signs of thalassemia is 90% for low-risk patients (class 1 Pesaro) and only 66-70% for those at high risk (class 3 Pesaro) according to EBMT data.

Thanks to the previous appropriate treatment, the little patient from Fundeni was classified in grade 1 (low) risk through a good transfusion policy. In Romania, the correct therapy with iron chelators was possible.

 

Bone marrow collection was performed in the ATI service and an operating room of the CC Iliescu Cardiology Institute. Along with the Romanian doctors came a renowned specialist from Israel, Prof. Dr Reuven, from the Hadassah University Hospital in Jerusalem.

The patient's post-transplant evolution is good, with a 100% graft, with complete normalization of the hemogram, which no longer makes the need for transfusion dependence; The minor and inherent complications of the two-month course after the transplant have been properly mastered, the doctors inform.

The success of a transplant depends on a multidisciplinary team that includes doctors, nurses, and nurses from the Spinal Transplant Department, along with numerous Laboratories and Clinics, the Hem biology and Transfusion Service of the Fundeni Clinical Institute.

Hematopoietic stem cell transplantation in thalassemia major TSCH

TSCH is the only therapy with curative potential in thalassemia major. The stem cell transplant procedure replaces the patient's bone marrow with the donor's, explains Dr Constantin Arion, from the Spinal Cord Transplant Center at the Fundeni Clinical Institute in Bucharest.

The first bone marrow transplant in a patient with thalassemia was performed in 1981.

·         A patient's chance of having a donor in the family is about 25%.

·         TCSH indications in thalassemia

·         Definitive indications:

·         transfusion-dependent thalassemia syndromes;

·         Age: under 16;

·         An HLA-compatible family donor.

·         Candidates who may be considered in special circumstances:

·         Thalassemia syndromes in patients 17-35 years;

·         Some cases of intermediate thalassemia.

·         Beta thalassemia (thalassemia)

 

Beta thalassemia is caused by the dysfunction of one or both genes that produce beta-globin. Man needs both globins, alpha and beta, to form haemoglobin. Beta thalassemia occurs mainly in the populations of the Mediterranean area (such as the Greeks or Italians) and less often in the population of Africa or Asia. There are several subtypes of beta-thalassemia, depending on the damage to the genes (only one or both of them are affected) and the level of gene production.

Types of thalassemia

 

If the patient has a genetic condition and has only one of the affected genes, he has mild anaemia and often does not need treatment. In this case, it is minor thalassemia or beta thalassemia country. If both globin genes are affected, the patient has severe anaemia. In this case, the patient inherited genes with thalassemia from both parents - thalassemia major. In the case of moderate anaemia (beta-thalassemia intermediate), blood transfusions may be needed. In the case of beta-thalassemia major, the symptoms of anaemia appear in the first six months of life. Doctor Muhammad Khan provides the best Clinical Consultation in the USA.  If blood transfusions start at an early age and continue throughout life, they will prolong life. Death occurs from damage to organs such as the heart or liver. Lack of oxygen loading and post-transfusion iron overload (hemochromatosis) lead to organ damage.

Beta thalassemia major is a rare congenital condition. There are about 350-400 patients in Romania with this condition (all these patients aged between 2 and 40 years. Repeated transfusions are the main treatment of patients with beta-thalassemia major and have proven to improve survival as well as hassle-free survival.