Aplastic anemia

Hematopoietic Stem Cell Transplantation

When
a Transplant
Is Needed
Results
of Stem Cell
Transplants
from Donors

The management of aplastic anemia primarily involves two approaches: immunosuppressive therapy and allogeneic hematopoietic stem cell transplantation. The choice of treatment depends on various factors, including the patient's age, the severity of the anemia, whether there's a suitable donor available, if there are associated conditions such as paroxysmal nocturnal hemoglobinuria (PNH), and the patient's general health and ability to tolerate treatment. Each of these factors plays a crucial role in deciding the most appropriate and effective treatment plan for the patient.

When is transplantation necessary? (Indications for transplantation)

Allogeneic hematopoietic stem cell transplantation (HSCT) does indeed have advantages over immunosuppressive therapy for those with severe aplastic anemia, including a shorter treatment duration and the potential for a long-term cure. Deciding on HSCT involves a personalized approach that considers the patient's age, the severity of the disease, and their suitability for undergoing a stem cell transplant. It's important that each patient's treatment plan is customized to maximize the chances

For patients younger than 50 who have a sibling with matching human leukocyte antigens (HLA), allogeneic hematopoietic stem cell transplantation is generally the recommended first treatment. This procedure involves transplanting healthy stem cells from a compatible sibling donor to replace the damaged bone marrow, offering the potential for a long-term cure of conditions like aplastic anemia.
For patients over 50 or for those who do not have an HLA-matched sibling donor, the first line of treatment is typically immunosuppressive therapy. If this approach does not yield successful results, the next step may involve a transplant using hematopoietic stem cells from an unrelated donor or a haploidentical family member, which means they are a partial HLA match. This alternative transplant option broadens the possibility for treatment even when a perfect match donor is not available.
In the past, allogeneic hematopoietic stem cell transplantation was a treatment option considered for a potential cure of paroxysmal nocturnal hemoglobinuria (PNH). Nowadays, with advancements in medical treatments and the introduction of new medications like C3 complement inhibitors, the reliance on transplantation for PNH alone has decreased. These drugs can effectively manage the disease, reducing the need for the more invasive transplantation procedure. However, if a patient has aplastic anemia in conjunction with PNH, stem cell transplantation remains a treatment consideration, following the same criteria as those for aplastic anemia itself.

▲ Korean Journal of Internal Medicine, Seung-Hwan Shin and Jong-Wook Lee, Recent advances in treatment of aplastic anemia, 2014

Hematopoietic Stem Cell Transplantation Outcomes at the Aplastic Anemia Center of the Hematology Hospital

Allogeneic hematopoietic stem cell transplantation is indeed a cornerstone of cellular therapy, a field at the forefront of contemporary medicine. The Aplastic Anemia Center at the Catholic Hematology Hospital stands out for its exceptional success rates, a testament to its vast experience. This excellence is recognized not just within the country but also when compared to prestigious international institutions.

For patients younger than 40 who have a matched human leukocyte antigen (HLA) donor, allogeneic hematopoietic stem cell transplantation has demonstrated excellent curative survival rates, often surpassing those seen in similar treatments at international centers. This treatment, which involves transplanting healthy stem cells from a compatible donor, can offer a potential cure for various hematologic diseases.

1)
Long-term survival rate at the Catholic Hematology Hospital Aplastic Anemia Center: 94% (based on 5 years, authored by Lee Jong-wook and Shin Seung-hwan, 2016)
2)
Long-term survival rate at major European institutions: 72% (based on 5 years, authored by Gupta et al., 2010)

Allogeneic hematopoietic stem cell transplantation, once limited to younger patients due to concerns about tolerance, has been revolutionized by new techniques. These advancements have opened the door for patients over the age of 40, who were previously thought to be unsuitable candidates, to receive this treatment. The application of these innovative methods is actively in progress and has been yielding results that are similar to the successful outcomes achieved in patients under 40 who have undergone transplantation. This is a significant milestone in the field, offering hope and expanded treatment possibilities to a broader age group.

1)
Long-term survival rate for patients under 40 years of age: 94% (based on 5 years, authored by Lee Jong-wook, Shin Seung-hwan, et al., 2016)
2)
Long-term survival rate for patients over 40 years of age: 89% (based on 5 years, authored by Lee Jong-wook, Shin Seung-hwan, et al., 2016)

In response to the limited availability of matched sibling donors for hematopoietic stem cell transplantation, the field has expanded to include alternative donor transplants. These come from unrelated donors or family members with half-matched HLA compatibility. The techniques for these alternative donor transplants, which are being actively employed at the Aplastic Anemia Center, have been honed to achieve outcomes that are favorable and often comparable to those of transplants with matched sibling donors. This progress significantly widens the potential for successful treatment for patients who previously might not have had suitable donors.

1)
Long-term survival rate for patients with sibling matched human leukocyte antigen allogeneic hematopoietic stem cell transplantation: 96% (based on 5 years, authored by Shin Seung-hwan, Lee Jong-wook, et al., 2018)
2)
Long-term survival rate for patients with unrelated donor hematopoietic stem cell transplantation: 88% (based on 5 years, authored by Shin Seung-hwan, Lee Jong-wook, et al., 2018)
3)
Long-term survival rate for patients with haploidentical family member allogeneic hematopoietic stem cell transplantation: 85% (based on 5 years, authored by Park Sung-soo, Lee Jong-wook, Park Sung-soo, et al., 2018)