Reviving a girl with severe immunodeficiency with stem cell transplant

At 18 months old, baby TL suffered from severe combined congenital immunodeficiency - the most severe form of congenital immune disorders - and overcame a challenging bone marrow stem cell transplant to be revived.

Children are closely cared for and monitored during the post-transplant period.

Baby girl has the most severe form of congenital immune disorder

Having a serious illness since 4 months after birth, baby girl TL (18 months old, in Hai Phong ) started having bouts of pneumonia, having to be hospitalized for long-term treatment. At this time, the child's immune system continuously encountered serious problems such as complications of swollen axillary lymph nodes after tuberculosis vaccination.

In August 2024, the family took the child to the National Children's Hospital for examination and performed in-depth immunological and genetic tests. The results determined that the child had severe combined congenital immunodeficiency due to a rare gene mutation.

Dr. Nguyen Thi Van Anh, Deputy Head of the Department of Immunology-Allergy-Arthritis, National Children's Hospital, said that TL has the most severe form of congenital immune disorder - Omenn syndrome, which has both severe lymphocytic cell deficiency making the child susceptible to severe infections and the body's auto-inflammatory response, causing the child to have red skin lesions all over the body, organ damage, and persistent elevation of liver enzymes.

In addition to isolation measures and the use of antibiotics against bacteria, fungi, tuberculosis, and viruses, children must also take immunosuppressants to reduce auto-inflammatory reactions. Therefore, preparations for the child's acute hematopoietic stem cell transplant are urgently carried out. This is an extremely important measure that can help the child's immune system recover completely.

TL can now enjoy playing and smiling like a healthy child.

After a hospital consultation chaired by Dr. Cao Viet Tung - Deputy Director of the National Children's Hospital, doctors determined that hematopoietic stem cell transplantation was the urgent way to save TL's life. However, this treatment phase was extremely stressful, because the child suffered from severe infection and liver damage, which seemed impossible to overcome.

With the determination of the doctors, through many consultations, multidisciplinary coordination of immunology, respiratory, hepatobiliary, resuscitation and stem cell, by July 2025 the child's general condition was more stable, ready for stem cell transplantation.

Overcoming the challenges of stem cell transplantation in children

Dr. Nguyen Ngoc Quynh Le, Head of the Stem Cell Transplantation Department, Stem Cell Center, National Children's Hospital, said that the stem cell transplant procedure in young children is particularly complicated. First, the patient must undergo a conditioning phase according to a specific chemical regimen, in order to remove all old bone marrow and prevent rejection.

For children under 1 year old, chemotherapy is very difficult, because their bodies are immature, have poor drug tolerance, and have a high risk of complications.

At the Stem Cell Center, bone marrow, after being taken from the patient's sister, is processed using a modern equipment system, ensuring strict sterile procedures. This process takes place very urgently and in less than an hour, the qualified stem cells are ready to be infused into the patient's body.

Directly performing the transplant, Dr. Nguyen Ngoc Quynh Le said that stem cell transplantation for young children is a huge challenge, especially for children with very severe infections like TL, requiring not only modern techniques but also close coordination between specialties: Respiratory resuscitation, nutrition, infection control, blood transfusion and immunology. Every step in the transplant room must be absolutely precise to ensure the safety of the child.

After the transplant, TL was treated in a completely sterile room, nourished intravenously, used new generation anti-rejection drugs, and closely monitored, with daily hematological and immunological tests.

There were times when the child suffered from severe pneumonia and blood infection and had to be supported by invasive mechanical ventilation and given many strong antibiotics to control the infection. However, with the relentless efforts and determination of the medical team during the care and resuscitation process, TL's new bone marrow cells developed, his blood parameters stabilized, and a new immune system was formed.

Up to now, TL can breathe on his own, breastfeed, laugh and play like a healthy baby and has been discharged from the hospital. The doctor assessed the baby's prognosis as positive, after returning to daily life, the child can completely develop normally like any other child.

The leaders and doctors of the Stem Cell Center happily saw the patient off the hospital.

Moved to see her child overcome the crisis and become healthy again, TL's mother choked up as she shared that this happiness cannot be expressed in words. "My family was given back by the doctors from the hands of death. The child being able to play and laugh again is a priceless gift. Our family is very grateful to the Hospital's Board of Directors, the doctors of the Stem Cell Center and the Department of Immunology-Allergy-Arthritis, who have always accompanied and helped my child and my family for nearly a year," the patient's mother emotionally said.

With the increasing incidence of congenital immunodeficiencies in children, most of which are not detected and treated promptly, TL's story is a testament to the importance of early diagnosis and the advancement of stem cell transplantation in medicine.

THIEN LAMB

Source: https://nhandan.vn/hoi-sinh-su-song-cho-be-gai-mac-suy-giam-mien-dich-nang-bang-ghep-te-bao-goc-post912770.html