The list of diseases treated with haematopoietic cells transplantation		  

          Despite the complications that the haematopoietic stem cells transplantation therapy currently bring, it is already used today in the treatment of more than fifty different terminal diseases, while the spectrum of these diseases is still enlarging (see the table). Some of them only occur rarely, some of them on the contrary, are among the most frequent causes of death (e.g. in a period between 3rd – 10th year of life, apart from injuries, leukaemia is the most common cause of death). During his life a man has a probability of more than 15 % of getting ill with some of these diseases. (However, haematopoietic stem cells transplant therapy is not suitable in every case.)

          These diseases can be divided to congenital and acquired. It has to be emphasized, that congenital diseases cannot be treated with own umbilical cord blood, because the haematopoietic stem cells located in it are also damaged. In such case the haematopoietic stem cells must be from the umbilical cord blood of siblings, and eventually from another source.

         Haematopoietic stem cell transplant therapy is limited by the fact that the matching source of haematopoietic stem cells can only be found for a percentage of patients. Even in case of such patients for whom a compatible donor can be found, the risk of death is very high due to incompatibility. Auto-transplant from umbilical cord blood assures certainty of compatible (matching) transplant (except congenital diseases, when the haematopoietic stem cells in umbilical cord blood are genetically damaged). As the umbilical cord blood is actually the blood of the child, it’s genetically identical with the child, and therefore the graft versus host reaction cannot develop. In addition, the haematopoietic stem cells in umbilical cord blood are very young and the storage keeps them young (as long as they are frozen they don’t actually age). Their vitality is higher than the vitality of the haematopoietic stem cells of adult donors. It can be supposed that in case of the patients who will have the auto-transplant from umbilical cord blood, the doctors will decide for transplantation far more often than now - while the severe complication can be expected by allogeneic transplantation.

          The haematopoietic stem cell transplantation is divided into autologous and allogeneic. We speak about autologous transplantation in case of transplantation of own haematopoietic cells from the own umbilical cord blood or from the own bone marrow. As the allogeneic transplantation it is understood transplantation, when the haematopoietic stem cells comes from sibling or from unrelated donor. The autologous transplantation from the umbilical cord blood could not be used in below listed diseases, which are genetically determined and in case of these, which origin reach the period before the birth. The own haematopoietic stem cells can be used only in such diseases, in which the risk of harvesting tumor cells is relatively low. The advantage of allogenous transplantation is, that it could be theoretically used in therapy of all below listed diseases. In practice however, in many diseases, the allogenous transplantation is not used due to the serious post-transplantation complications.

          It is generally valid, that the therapy selection depends on many different factors. Hence the choice of treatment in case of the concrete patient is highly individual.

At the end of a list we present some diseases, in which it could be possible with the highest probability to use the stem cells from umbilical cord blood in the future.
Current application of haematopoetic stem cells
         
Disease Autologous cord blood Cord blood from a sibling Autologous bone marrow Bone marrow from a donor
Acute Leukemias        
Acute Lymphocytic Leukemia yes - yes yes - yes
Acute Myelogenous Leukemia yes yes yes yes
Chronic Leukemias        
Chronic Lymphocytic Leukemia yes yes yes - yes
Chronic Myelogenous Leukemia yes yes yes - yes
Juvenile Myelomonocytic Leukemia yes yes yes - yes
Myelodysplastic
Syndromes		        
Chronic Myelomonocytic Leukemia yes yes no yes
Refractory Anemia yes yes yes - yes
Refractory Anemia with Excess Blasts yes yes yes - yes
Refractory Anemia with Excess Blasts in Transformation yes yes yes - yes
Refractory Anemia with Ringed Sideroblasts yes yes yes - yes
Stem Cell Disorders        
Severe Aplastic Anemia yes yes nie yes
Congenital Cytopenia no yes no yes
Dyskeratosis Congenital no yes no yes
Fanconi Anemia no yes no yes
Paroxysmal Nocturnal Hemoglobinuria yes yes no yes
Myeloproliferative
Disorders		        
Acute Myelofibrosis yes yes no yes
Agnogenic Myeloid Metaplasia yes yes no yes
Lymphoproliferative
Disorders		        
Hodgkin's Disease yes yes yes ±
Non-Hodgkin's Lymphoma yes yes yes ±
Plasma Cell Disorders        
Multiple Myeloma yes yes yes - ±
Plasma Cell Leukemia yes yes yes - ±
Other Malignancies        
Breast Cancer (yes) (yes) (yes) exp
Ovarian Cancer (yes) (yes) (yes) exp
Small-Cell Lung Cancer (yes) (yes) (yes) exp
Brain Tumors yes (yes) yes no
Ewing Sarcoma yes (yes) yes exp
Neuroblastoma yes yes yes ±
Renal Cell Carcinoma (yes) (yes) (yes) yes/exp
Testicular Cancer yes yes yes exp

Autoimmune Diseases

        
Evan Syndrome yes yes exp no
Multiple Sclerosis yes yes yes - exp
Rheumatoid Arthritis yes yes yes - exp
Systemic Lupus Erythematosus yes yes yes - exp

Phagocyte Disorders

        
Chediak-Steinbrick-Higashi Syndrome no yes no yes
Chronic Granulomatous Disease no yes no yes
Neutrophil Actin Deficiency no yes no yes
Reticular Dysgenesis no yes no yes

Liposomal Storage
Diseases

        
Adrenoleukodystrophy no yes no yes
Gaucher's Disease no yes no yes
Hunter's Syndrome no yes no yes
Hurler's Syndrome no yes no yes
Krabbe Disease no yes no yes
Maroteaux-Lamy Syndrome no yes no yes
Metachromatic Leukodystrophy no yes no yes
Morquio Syndrome no yes no yes
Mucolipidosis II no yes no yes
Mucopolysaccharidoses no yes no yes
Nomann-Pick Disease no yes no yes
Sanfilippo Syndrome no yes no yes
Scheie Syndrome no yes no yes
Sly Syndrome, Beta-Glucuronidase Deficiency no yes no yes
Wolman Disease no yes no yes

Histiocytic Disorders

        
Familial Erythrophagocytic Lymphohistiocytosis no yes no yes
Hemophagocytosis no yes no yes
Histiocytosis-X no yes no yes
Langerhans' Cell Histiocytosis no yes no yes

Inherited
Erythrocyte
Abnormalities

        
Beta Thalassemia Major no yes no yes
Blackfan-Diamond Anemia no yes no yes
Pure Red Cell Aplasia no yes no yes
Sickle Cell Disease no yes no yes

Congenital (Inherited)
Immune System
Disorders

        
Absence of T & B Cells no yes no yes
Absence of T Cells no yes no yes
Ataxia-Telangiectasia no yes no yes
Bare Lymphocyte Syndrome no yes no yes
Common Variable Immunodeficiency no yes no yes
DiGeorge Syndrome no yes no yes
Kostmann Syndrome no yes no yes
Leukocyte Adhesion Deficiency no yes no yes
Omenn's Syndrome no yes no yes
Severe Combined Immunodeficiency (SCID) no yes no yes
SCID with Adenosine Deaminase Deficiency no yes no yes
Wiskott-Aldrich Syndrome no yes no yes
X-Linked Lymphoproliferative Disorder no yes no yes

Other Inherited
Disorders

        
Cartilage-Hair
Hypoplasia		 no yes no yes
Ceroid Lipofuscinosis no yes no yes
Congenital Erythropoietic Porphyria no yes no yes
Glanzmann Thrombasthenia no yes no yes
Lesch-Nyhan Syndrome no yes no yes
Osteopetrosis no yes no yes
Tay Sachs Disease no yes no yes

Inherited Platelet
Abnormalities

        
Amegakaryocytosis / Congenital Thrombocytopenia no yes no yes

Potential Future Stem Cell Applications
Alzheimer's Disease yes no yes no
Diabetes yes no yes no
Heart Disease yes no yes no
Liver Disease yes no yes no
Muscular Dystrophy yes no yes no
Parkinson's Disease yes no yes no
Spinal Cord Injury yes no yes no
Stroke yes no yes no

 

Vysvetlivky:

yes - in cases of umbilical cord blood it is possible to perform the transplantation and its possible to expect a recovery
- in cases of bone marrow the transplantation is performed in a standard way, mostly with curative effect
yes- the transplantation is carried out, but it is impossible to expect lasting recovery from it, only prolongation of survival
(yes) the transplantation could be performed, nowadays it is however, by the given diagnosis, only a subject of the research, the real importance will be shown in the future
± in some cases the transplantation is performed, the permanent recovery could be expected, but because of the high risk of such intervention it is not a standard method
exp it is only an experimental method

   UMBILICAL CORD BLOOD UTILISATION
   : Umbilical cord blood
   : Stem cells
   : Haematopoietic cells
   : Alternative umbil. cord blood utilisation
   : The probability of using the transplant
   : Alternatives to own umbilical cord blood
   UMBILICAL CORD BLOOD HARVESTING
   UMBILICAL CORD BLOOD PROCESSING
   COMPARING WITH THE COMPETITION
   : Umbilical cord blood harvesting
   : The umbilical cord blood transport
   : The umbilical cord blood processing
   : The storage