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Advancements in the Treatment of Blood Disorders

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Today, blood disorders are curable in a very high percentage of cases, and the majority of those not yet curable are still manageable in the long term. In recent years, significant progress has been made for diseases suchas acute and chronic leukemias, multiple myeloma, Hodgkin’s lymphoma, and non-Hodgkin’s lymphoma.

We asked Prof. Angelo Carella, a specialist in hematology at Paideia International Hospital, to share these advancements.

 

Hematological Neoplasms

In the last 20 years, explains Prof. Carella, we have witnessed tremendous progress in the biology and therapy of hematological neoplasms. Today, they are called “targeted and intelligent therapies“: the so-called “targeted therapies.” These innovative therapies have already allowed patients with acute promyelocytic leukemia to recover in 90% of cases, whereas just afew years ago, survival for this disease was less than 10%.

Chronic myeloid leukemia, thanks to these selective drugs, has become a condition that the vast majority of patients can live with. Much more recent are the enormous therapeutic advances for multiple myeloma: thanks to new drugs that act on the mechanisms that cause myeloma cells to proliferate, complete remission of the disease is also possible.

 

The NewCar-T Therapy

The fight against blood cancers has not stopped at these progressions, Prof. Carella continues, but is making giant strides towards precision medicine, increasingly chemo-free in many cases, based on intelligent drugs to target only diseased cells, drastically reducing toxicity. A procedure with great prospects is the so-called Car-T therapy. It involves extracting the patient’s lymphocytes and arming them in the laboratory so that they can target a marker present on leukemia, lymphoma, or myeloma cells, and then infusing them back into patients with the hope that these cells can not only identify but also kill the tumor cells. Important results have already been achieved, including healing a very high percentage of children and adults considered refractory or relapsed multiple times with acute lymphoblastic leukemia or aggressive non-Hodgkin lymphomas.

 

Hodgkin’s and Non-Hodgkin’s Lymphomas

Even in Hodgkin’s lymphoma, where we already had effective drugs, protocols have been developed using new, selective drugs that offer very high complete remissions, even in relapsed patients. Regarding large B-cell non-Hodgkin lymphomas, significant progress has been recorded with two new drugs that increase complete remissions. An additional possibility is offered by the so-called bispecific antibodies, at least two of which are gaining prominence in advanced patients. These antibodies are proving to be very important in relapsed patients with multiple myeloma.

They will certainly be used earlier in the near future.

In chronic myeloproliferative syndromes (polycythemia, thrombocythemia, and myelofibrosis), cellular biology and mutational studies have provided important knowledge that has allowed the development of fairly selective drugs, especially in myelofibrosis, a more severe condition than others, where allogeneic transplantation is essential for patients under 65 years old to achieve healing.

At the moment, acute myeloid leukemia is lagging behind in results, although biology has made enormous progress and has explained the fine mechanisms underlying the leukemic process.

It has also highlighted how there is no acute myeloid leukemia but rather many different forms at the molecular level. Recently, good results have been obtained, especially in the treatment of elderly (but also young) individuals with the combination of two drugs (Azacitidine or Decitabine and Venetoclax). However, the only real cure comes from allogeneic transplantation, which should be performed when the patient is in remission after chemotherapy.

Finally, chronic lymphocytic leukemia is a very common condition that often does not require therapy except for patients with very aggressive genetic mutations; for this group, numerous selective drugs are available, proving to be particularly effective.

The new therapies, mostly biological and molecular, have completely revolutionized the approach to most blood cancers, but we are only at the beginning; surely, in the coming years, we may even achieve a cure for many of these diseases.

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