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Successful Bone Marrow Stem Cell Transplant: Reviving Life for a Baby Girl with Severe Combined Immunodeficiency

Diagnosed with a severe illness at just 4 months old, baby T.L (18 months old, from Hải Phòng) faced a challenging journey after being diagnosed with severe combined immunodeficiency (SCID) – one of the most serious congenital immune disorders. Without a hematopoietic stem cell transplant, she was at high risk of death within her first year of life. The successful transplant at Vietnam National Children’s Hospital not only saved her life but also showcased the scientific and technical advancements of pediatric medicine in Vietnam.

Baby T.L with Severe Combined Immunodeficiency Given a New Lease on Life

From recurrent pneumonia to diagnosis of a rare disease

According to the family, when T.L was 4.5 months old, she began experiencing recurrent pneumonia and had to be hospitalized for long-term treatment. At this time, her immune system was continuously failing. Later, she developed swollen lymph nodes under her arm following a tuberculosis vaccination. In August 2024, the family brought her to Vietnam National Children’s Hospital for examination. There, advanced immunology and genetic tests confirmed that she had severe combined immunodeficiency caused by a rare gene mutation.

Dr. Nguyễn Thị Vân Anh, Deputy Head of the Department of Immunology – Allergy – Rheumatology at Vietnam National Children’s Hospital, said: “T.L suffers from the most severe form of congenital immune disorder – Omenn syndrome – characterized by a severe reduction in lymphocyte cells, making her highly susceptible to severe infections. At the same time, her body showed autoimmune-like inflammatory reactions causing extensive skin damage, organ injuries, and persistent liver enzyme elevations. Besides isolation and antibiotics against bacteria, fungi, tuberculosis, and viruses, she needed immunosuppressive drugs to control these autoimmune responses. Therefore, preparing for an urgent hematopoietic stem cell transplant was critical as it could fully restore her immune system.”

After a hospital consultation chaired by Dr. Cao Việt Tùng – Deputy Director of Vietnam National Children’s Hospital, doctors agreed that a hematopoietic stem cell transplant was the essential life-saving treatment for T.L. However, this phase was extremely challenging because she had severe infections and liver damage that seemed insurmountable. Thanks to the dedication of the medical team and multiple multidisciplinary consultations involving immunology, respiratory, hepatology, intensive care, and stem cell experts, by July 2025 her condition stabilized enough to proceed with the transplant.

Stem Cell Transplant – A Lifesaving Opportunity for the Patient

Stem cell transplantation in young children is particularly complex. The patient must first undergo conditioning chemotherapy to eliminate the old bone marrow and prevent rejection. For infants under one year old, chemotherapy is very difficult due to their fragile bodies, poor drug tolerance, and high risk of complications.

At the Stem Cell Center, bone marrow stem cells harvested from the patient’s older sister were processed using advanced equipment under strict sterile conditions. This urgent process took less than an hour to prepare the transplant-ready stem cells for infusion.

The external cell separation system using magnetic technology and modern storage and processing equipment at the Stem Cell Center – Vietnam National Children’s Hospital

Dr. Nguyễn Ngọc Quỳnh Lê – Head of the Stem Cell Transplant Department at the Stem Cell Center, Vietnam National Children’s Hospital, who performed the transplant, shared: “Stem cell transplantation in young children is a great challenge, especially for patients like T.L with severe infections. It requires not only advanced technology but also close cooperation across specialties – respiratory intensive care, nutrition, infection control, transfusion, and immunology. Every step in the transplant room must be precisely accurate to ensure patient safety.”

TS.BS Nguyễn Ngọc Quỳnh Lê – Trưởng khoa Ghép Tế bào gốc, Trung tâm Tế bào gốc, Bệnh viện Nhi Trung ương xúc động chia sẻ về thành công của ca ghép

After the transplant, T.L was treated in a completely sterile room, nourished intravenously, given next-generation anti-rejection drugs, and closely monitored daily through hematology and immunology tests. During some stages, she suffered from severe pneumonia and sepsis, requiring mechanical ventilation and strong antibiotics to control infections. Despite these challenges, thanks to the relentless efforts of the medical team in care and intensive therapy, T.L’s new bone marrow grew successfully, blood counts stabilized, and a new immune system developed.

Close monitoring and care of the patient during the post-transplant period

Nearly One Year of Courageous Treatment, Baby Girl Overcomes the Brink of Death

Now, T.L can breathe independently, breastfeed, smile, and play like a healthy child and has been discharged from the hospital. Doctors assess a positive prognosis, and after returning home, she is expected to develop normally like other children.

Now T.L can freely play and smile like a healthy child

T.L’s mother, overwhelmed with emotion seeing her child survive and regain health, shared: “This happiness is beyond words. The doctors gave us our child back from the hands of death. Being able to see her play and smile again is an invaluable gift. Our family is deeply grateful to the hospital leadership, the doctors at the Stem Cell Center, and the Department of Immunology – Allergy – Rheumatology who have stood by and supported us throughout this almost one-year journey.”

Hospital leaders and medical staff from the Stem Cell Center joyfully see off the patient upon discharge

Stem Cell Transplant – An Important Step Forward in Modern Medicine

The success of the transplant for baby T.L not only saved a precious life but also affirmed the professional expertise, multidisciplinary collaboration, and ability to apply advanced and specialized techniques of Vietnam National Children’s Hospital in the field of hematopoietic stem cell transplantation.

Given the rising number of congenital immunodeficiency cases in children, many undiagnosed and untreated timely, T.L’s story highlights the importance of early diagnosis and advances in stem cell transplantation. This success motivates the medical team at Vietnam National Children’s Hospital to continue researching, developing, and improving techniques to offer more life-saving opportunities to children in the future.

Information and Communications Department
Institute of Education and Research on Health and Education

Category: Medical News

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