Using the hematopoetical STEM cells from cord blood is something new. They are mainly used as an alternative to the hematopoetic bone marrow which contains adult hematopoetic STEM cells..
Diseases treated with hematopoietic stem cell transplantation:
The first hematopoietic stem cell transplantation performed successfully was achieved 42 years ago (1968), by Dr. Robert A. Good, University of Minnesota. A new therapy age was thus opened for severe hematological diseases that had been considered as incurable until then. Current standard indications for performing a hematopoietic stem cell transplantation include over 70 diseases, most of them malignant and non malignant hematological diseases, but also certain genetic diseases or malignant solid tumors.
Hematopoietic stem cells existing in the bone marrow are the resource of hematopoiesis. Both hematopoietic stem cells and the progenitors (intermediate stages of cell specialization) under the influence of various factors (e.g. genetic factors, environmental factors, infections, etc.) may be affected, resulting in a number of diseases.
These factors can work towards stimulating uncontrolled proliferation of cells belonging to one or more blood cell lines (hematological malignancies e.g. Leukemia) or towards inhibiting the activity of bone marrow hematopoiesis (stem cell diseases with bone marrow failure e.g. Aplastic anemia ).
Another category of non-malignant diseases with indication of making a stem cell transplant, due to severity of evolution and prognosis are hereditary blood diseases affecting the quality or function of blood cells (e.g. in hemoglobinopathies red blood cells are affected; in chronic granulomatosis- neutrophils in immune deficits are affected – white blood cells affected populations) and hereditary metabolic diseases (e.g. Gaucher's disease, mucolipidosis).
For these affections the hematopoietic stem cells graft has a curative, healing role, in some cases being the only saving therapy means. Hematopoietic stem cell transplantation (blood cell maker) is to replace diseased cells with healthy cells after the former were destructed by prior chemoradiotherapy.
Of the category of standard indications of stem cell transplantation, leukemia is the most common malignancy of children, representing about 1 / 3 of the total number of cases of malignancy found in children, followed by brain tumors and Hodgkin's disease.
In Romania it is estimated that 1,500 children suffer from leukemia, acute leukemia representing 10% of common cancers in this age category and the leading cause of death in 35 years. (Acute lymphoblastic leukemia is the most common hematological malignancy arising in childhood, about 80-90% of cases appear under the age of 10 years).
Add to these, 3500 patients with Hodgkin's disease, some of these having the indication to undergo a hematopoietic stem cell transplantation for survival and 3.700 children with thalassemia major, an inherited blood disease in which the only cure is the transplantation of hematopoietic stem cells from a healthy donor.
Stem cell transplantation may be autologous or allogeneic.(see details above). If the disease has genetic determinism (is caused by an alteration of genetic information), only allogeneic transplantation (transplantation from another healthy, immunologically compatible individual) which should bring "healthy” genetic information (e.g. immunological congenital deficits or congenital metabolic diseases). can play a curative "healer" role.
We know a large number of such diseases (see table), but they have an incidence (frequency) very small population. But if the disease has no such genetic determinism, both autologous transplantation and allogeneic transplantation may be curative role.
Stem cell transplantation may also play only the adjuvant role (complementary) to the fund treatment of some solid malignant tumors. In these cases, the availability of a stored graft and the chance to perform the stem cell transplant allows a therapeutic reassessment of the patient.
Thus it is possible to apply a more aggressive anti-tumor treatment, compared to the usual one (with higher doses of chemo / radiotherapy), aiming to reduce metastasis or tumor recurrence.
This aggressive treatment is accompanied by bone marrow toxic effects (destruction of bone marrow cells); therefore further administration of a hematopoietic stem cell grafts, which should thus provide bone marrow cell repopulation (e.g. malignant solid tumors: neuroblastoma, retinoblastoma) is required.
|
Disease |
Autolog Transplant |
Alogen Transplant |
|
ACUTE LEUKEMIAS |
|
|
|
Acute Lymphoblastic Leukemia (ALL) |
Yes * |
Yes |
|
Acute Myelogenous Leukemia (AML) |
Yes |
Yes |
|
Acute Biphenotypic Leukemia |
Yes |
Yes |
|
Acute Undifferentiated Leukemia |
Yes |
Yes |
|
CHRONIC LEUKEMIAS |
|
|
|
Chronic Myelogenous Leukemia (CML) |
Yes # |
Yes |
|
Chronic Lymphocytic Leukemia (CLL) |
Yes |
Yes |
|
Juvenile Chronic Myelogenous Leukemia (JCML) |
Yes # |
Yes |
|
Juvenile Myelomonocytic Leukemia (JMML) |
Yes # |
Yes |
|
LYMPHOPROLIFERATIVE DISORDERS |
|
|
|
Hodgkin’s Disease |
Yes |
Yes |
|
Non Hodgkin’s Lymphoma |
Yes |
Yes |
|
Burkitt’s Lymphoma |
Yes |
Yes |
|
MYELODYSPLASTIC SYNDROMES |
|
|
|
Refractory Anemia (RA) |
Yes# |
Yes |
|
Refractory Anemia with Excess Blasts (RAEB-T) |
Yes# |
Yes |
|
Refractory Anemia with Ringed Sideroblasts(RARS) |
Yes# |
Yes |
|
Refractory Anemia with Excess Blasts |
Yes# |
Yes |
|
Chronic Myelomonocytic Leukemia (CMML) |
Yes# |
Yes |
|
MIELOPROLIFERATIV DISEASES |
|
|
|
Myeloid Metaplasia |
Yes # |
Yes |
|
Agnogenic Myeloid Metaplasia with Myelofibrosis |
Yes # |
Yes |
|
MONOCLONAL GAMMOPATHY |
|
|
|
Multiple Myeloma |
Yes @ |
Yes |
|
Plasma cell leukemia |
Yes @ |
Yes |
|
Waldestrom ‘s Macroglobulinemia |
Yes @ |
Yes |
|
|
|
|
|
INHERITED ERYTHROCYTE ABNORMALITIES |
|
|
|
Beta Thalassemia Major |
No |
Yes |
|
Blackfan-Diamond Anemia |
No |
Yes |
|
Pure Red Cell Aplasia |
No |
Yes |
|
Sickle Cell Disease |
No |
Yes |
|
STEM CELL DISORDERS |
|
|
|
Aplastic Anemia (severe) |
Yes ! |
Yes |
|
Congenital Cytopenia |
No |
Yes |
|
Congenital Dyskeratosis |
No |
Yes |
|
Fanconi Anemia |
No |
Yes |
|
Paroxysmal Nocturnal Hemoglobinuria (PNH) |
Yes |
Yes |
|
PHAGOCYTE DISORDERS |
|
|
|
Chediak-Higashi Syndrome |
No |
Yes |
|
Chronic Granulomatous Disease |
No |
Yes |
|
Reticular Dysgenesis |
No |
Yes |
|
Neutrophil Actin Deficiency |
No |
Yes |
|
LIPOSOMAL STORAGE DISEASES |
|
|
|
Adrenoleukodystrophy |
No |
Yes |
|
Gaucher’s Disease |
No |
Yes |
|
Hunter ‘s Syndrome (MPS –II) |
No |
Yes |
|
Hurler’s Syndrome (MPS- IH) |
No |
Yes |
|
Krabbe Disease |
No |
Yes |
|
Maroteaux-Lamy Syndrome (MPS – VI) |
No |
Yes |
|
Metachromatic Leucodystrophy |
No |
Yes |
|
Morquio Syndrome (MPS – IV) |
No |
Yes |
|
Mucolipidosis II (I – cell disease) |
No |
Yes |
|
Niemann-Pick Disease |
No |
Yes |
|
San Filipp Syndrome (MPS III) |
No |
Yes |
|
Scheie Syndrome (MPS – IS) |
No |
Yes |
|
Sly Sindrome (MPS – VII) |
No |
Yes |
|
Wolman Disease |
No |
Yes |
|
HISTIOCYTIC DISORDERS |
|
|
|
Familial erythrophagocytic lymphohistiocytosis |
No |
Yes |
|
Hemophagocytosis |
No |
Yes |
|
Histiocytosis X |
No |
Yes |
|
Langerhans ‘Cell Histiocytosis |
No |
Yes |
|
CONGENITAL (INHERITED) IMMUNE SYSTEM DISORDER |
|
|
|
Absence of T or B cells |
No |
Yes |
|
Ataxia Teleangiectasia |
No |
Yes |
|
Bare Lymphocyte Syndrome |
No |
Yes |
|
Common Variable Immunoficiency |
No |
Yes |
|
DiGeorge Syndrome |
No |
Yes |
|
Kostmann Sindrome |
No |
Yes |
|
LeuKocyte Adhesion Deficiency |
No |
Yes |
|
Omenn’s Syndrom |
No |
Yes |
|
Severe Combined Immunodeficiency (SCID) |
No |
Yes |
|
SCID with Adenosine Deaminase Deficiency |
No |
Yes |
|
Wiskott-Aldrich Syndrome |
No |
Yes |
|
X Linked Lymphoproliferative Disorder |
No |
Yes |
|
OTHER INHERITED DISORDER |
|
|
|
Ceroid Lipofuscinosis Congenital |
No |
Yes |
|
Porphyria Erythropoietic |
No |
Yes |
|
Glanzmann Thrombasthenia |
No |
Yes |
|
Lesch-Nyhan Syndrome |
No |
Yes |
|
Osteopetrosis |
No |
Yes |
|
Tay Sachs Disease |
No |
Yes |
|
Sandhoff Disease |
No |
Yes |
|
INHERITED PLATELET ABNORMALITIES |
|
|
|
Amegakaryocytosis |
No |
Yes |
|
Congenital Thrombocytopenia |
No |
Yes |
|
AUTOIMMUNE DISEASES |
|
|
|
Evans Syndrome |
Yes |
Yes |
Legend:
Yes: Using cord blood cell transplant can cure the disease
No: Transplant cannot be made
Yes*: Cord Blood cell transplant used only in cases of some sub-types of diagnosys. Alogen transplant is prefered if available
Yes& : in case of diagnosys sub-types with great risks, alogen transplant is prefered if available
Yes#: Using alogen transplant in case of adult diseases has limited results. There are no results regarding autolog transplantation yet. Alogen transplant is prefered
Da@: the transplant is usable but there is no cure only extending life expectation
Da! : in case of failure of bone marrow transplant, an alogen one is prefered
Adjuvant Therapy
|
Disease |
Autolog Transplant |
Alogen Transplant |
|
Central Nervous System Tumors |
Yes |
Yes (experiment) |
|
Ewing’s Sarcom |
Yes |
Yes (experiment) |
|
Retinoblastoma |
Yes |
Yes |
|
Neuroblastoma |
Yes |
Yes |
|
Testicular Cancer |
Yes |
Yes |
Clinical trial therapies
|
Malignant tumors (adjuvant therapy) |
Breast cancer |
|
|
|
|
Central Nervous System Diseases |
Multiple sclerosis |
|
Other Inherited Immune system disorder |
Boala Gunther (porfiria eritropoetica) |
| Gene Therapy |
Glanzmann Thrombasthenia Severe combined Immunodeficiency |
Experimental therapy
|
Autoimmune diseases |
Juvenile Rheumathoid Arthritis |
|
Terapie reparatorie neuronala |
Central Nervous System Diseases
Post cerebral stroke |
|
Organic Regenerative Therapy |
Renal regenerative therapy by asociated renal transplant and stem cell transplant Liver – Regenerative Therapy |
|
Gene Therapy |
Fanconi Anemia |