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HomeHealth articlesstem cell transplantWhat Are the Non-malignant Disorders for Which Hematopoietic Stem Cell Therapy Is Advocated?

Hematopoietic Stem Cell Transplantation for Non-malignant Disorders

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Hematopoietic stem cell transplantation is a potent therapeutic choice for non-malignant disorders. Read the article to know more about it.

Medically reviewed by

Dr. Abdul Aziz Khan

Published At May 24, 2024
Reviewed AtMay 24, 2024

Introduction:

The concept of hematopoietic stem cell transplantation was validated and began in the 1950s, following the outcome yielded through an observational animal study. By 1957, the medical history underscored the first-ever successful instance of hematopoietic stem cell transplantation. Since then, hematopoietic stem cell transplantation has been designated as a potent and appreciable therapeutic line for tackling a wide spectrum of ailments. Till now, it has been perceived as a hot topic of discussion owing to various therapeutic attributes that it could bring forth.

What Is Hematopoietic Stem Cell Transplantation?

Hematopoietic stem cell transplantation is a peculiar stem cell therapy where the healthy hematopoietic stem cells (forebearers of blood cells) are permeated into the streaming circulation. These hematopoietic stem cells could be transfigured into active and functional blood cells. Bone marrow, peripheral blood, and umbilical cord are the reservoirs of hematopoietic stem cells.

In hematopoietic stem cell transplantation, the hematopoietic stem cells that are extracted from either the patient’s body or from another person who exhibits acceptable compatibility with the patient’s blood type and other tissue proteins are instituted for transplantation. Allogeneic and autologous hematopoietic stem cell transplantation are the two discrete stem cell transplantation modalities where the donor for the former one is another person while that of the latter is one’s own body. Similarly, the location from which the hematopoietic stem cell ought to be harvested needs to be strategically mapped out.

The hematopoietic stem cells garnered and harvested from the designated sites are then infused and phased into the bloodstream. The most appropriate and desirable vein is punctured and accessed to infuse in the hematopoietic stem cells. The infused stem cells showcase an expedited transfiguration into functional blood cells, which cover up the derangements or depletion in the blood profile inflicted by certain ailments or illnesses. Thus, hematopoietic stem cell therapy palliates the derangement in the blood profile inflicted by underlying disease conditions.

What Are the Non Malignant Disorders for Which Hematopoietic Stem Cell Therapy Is Advocated?

The critical field of applicability concerning hematopoietic stem cell transplantation is for blood cell cancers. This technique strengthens and deepens the proportion of healthy blood cells, which, when assisted by appropriate anticancer modalities like chemotherapy or radiation therapy, augment the cancer cell suspension while upscaling the healthy blood cell count. In addition to blood cell cancers, hematopoietic stem cell transplantation is also advocated for certain other ailments. Here are a few of the non-malignant (diseases with the ability to stretch out to other bodily parts) medical conditions where hematopoietic stem cell therapy is being advocated:

  • Aplastic Anemia: Aplastic anemia is prompted by the disablement of the bone marrow, which eventuates momentous downfall in the blood cell proportions. It could bring forth dizziness, fatigue, pale skin, shortness of breath, and a heightened inclination for bleeding. Concerning aplastic anemia, hematopoietic stem cell transplantation exhibited gravitated treatment outcome when equated with that of the conventional therapy. Retrospective and systematic scientific studies on aplastic anemia underscored the above conclusion.

  • Thalassemia: Thalassemia is brought on by downturned hemoglobin (a proteinaceous entity in the red cell that renders the potentiality for holding the oxygen molecule) formation. The hemoglobin formation is guided and mastered by certain genetic components (hemoglobin genes). In thalassemia, the genetic integrant concerning the hemoglobin gets altered (mutated), engendering derangements in the hemoglobin microstructure. Hematopoietic stem cell transplantation in thalassemia patients showcased appreciable treatment outcomes with a marked upswing with respect to longevity.

  • Severe Combined Immune Deficiency Syndrome: Severe combined immune deficiency syndrome is a peculiar medical state brought on by errors in the corresponding genes that supervise and tune the white blood cells. It is white blood cells that render immunity and extend defense against infections and microbial invasions. Depleted white blood cell proportions are encountered with severe combined immune deficiency syndrome. Hematopoietic stem cell transplantation augmented the survival prospects in severe combined immune deficiency syndrome. The earlier the transplantation is instituted, the better the treatment outcome yielded.

  • Sickle Cell Disease: In sickle cell disease, the genetic integrant that governs the hemoglobin microstructure is deranged. Owing to the aberration in the genetic counterpart, the red blood cells, which are otherwise disk-shaped, reflect a sickle-shape (C-shaped). Sickle shape gravitates the occlusion and plugging of the blood vessel; these sickled red cells die out and perish prematurely. In such cases, appreciable outcomes are expressed with allogeneic hematopoietic stem cell transplantation.

  • Fanconi Anemia: Another genetically guided ailment is Fanconi anemia, where the proportions of all the blood cells express remarkable depletion. The red blood cell, platelet, and white blood cell proportions palliate down in Fanconi anemia, reflecting a gross bone marrow disablement. Hematopoietic stem cell transplantation is being advocated for Fanconi anemia to reinstate the blood cell proportions.

  • Chediak-Higashi Syndrome: Chediak-higashi syndrome undermines the immune cells, blunting and muting their defensive attributes. The disabled and debilitated immune functions escalate one’s propensity for infections. Hematopoietic stem cell transplantation could be advocated in Chediak-higashi syndrome to overhaul and reinstate the functional state of the immune cells.

  • Leukocyte Adhesion Deficiency: In the leukocyte adhesion deficiency condition, the white blood cells (leukocytes) are incapacitated in invoking defense against microbial influx and invasion. This condition encourages one’s prospect for infectious ailments owing to undermined immune functions instigated by the leukocyte adhesion deficiency. Hematopoietic stem cell transplantation subjugates the palliated immune response by rendering healthy white blood cells.

  • Chronic Granulomatous Disease: In chronic granulomatous disease, a specific white blood cell, so-called phagocytes, are functionally muted and impaired. Phagocytes are integrants for immune functions, which contribute by eating up the disease-invoking agents in the blood. The eradication of the pathogen expresses challenges owing to phagocyte impairment. Hematopoietic stem cell transplantation yields functional phagocytes, thus reinstituting immunity and microbial defense.

  • Kostmann Syndrome: Kostmann syndrome is a peculiar inborn ailment with alarming palliation of the neutrophil (a subset of white blood cells) proportions. Neutrophils, being the key immune component, are concerned with one’s immune state. Remarkable mitigation in the neutrophil count heightens the scope for infections and other microbial ailments. Hematopoietic stem cell transplantation upscales the neutrophil proportion in Kostmann syndrome and thereby buttresses the immune functions.

Conclusion:

Hematopoietic stem cell transplantation has been appreciated and recognized for tackling a wide spectrum of ailments. Though its prime area of therapeutic application is for malignant conditions, it could also bring forth surprising outcomes in non-malignant conditions as well. Different ailments that invoke derangements and upsets in the blood profile could be tackled with hematopoietic stem cell transplantation. However, the candidate for hematopoietic stem cell transplantation ought to be nominated strategically to repress and subdue the risk quotient and to harvest the best outcome.

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Dr. Abdul Aziz Khan
Dr. Abdul Aziz Khan

Medical oncology

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