Symptoms of rare blood cancer in children: Paedriatic oncologist says why bone marrow transplants are crucial

How to cure Juvenile Myelomonocytic Leukemia: The most effective treatment for JMML is an allogeneic hematopoietic stem cell transplant, better known as a bone marrow transplant (BMT), say experts (Image: Pexels)

Juvenile Myelomonocytic Leukemia, or JMML, is a rare blood cancer that afflicts toddlers. It is aggressive and can spread quickly if untreated. However, with the right approach, particularly bone marrow transplantation, children have a chance at survival.

Causes of JMML

According to Dr Satya Prakash Yadav, Senior Director of Paediatric Bone Marrow Transplant, Medanta, Gurugram, JMML is caused by tiny changes, called mutations, in a child’s DNA. “Genes like PTPN11, NF1, CBL, NRAS, and KRAS, which normally guide cell growth, go a little haywire. These mutations are almost always acquired during the child’s lifetime, they’re not usually inherited from parents,” Dr Yadav tells Moneycontrol. The result is an overproduction of abnormal monocytes, a type of white blood cell, which crowd out healthy cells and can damage vital organs.

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Symptoms of JMML

Dr Yadav says JMML can be tricky to detect because early symptoms are usually same as common childhood illnesses:

• Persistent fever that won’t respond to typical treatment
• Skin rashes that resemble eczema
• Pale skin, easy bruising, or unusual bleeding
• Frequent infections
• Swollen belly from enlarged liver and spleen

Blood tests revealing high white cell counts, particularly monocytes, low red cells, and low platelets usually raise suspicion. “To confirm JMML, we perform a bone marrow biopsy and genetic tests to identify the specific mutations,” says Dr Yadav.

Standard chemotherapy alone rarely cures JMML. The most effective treatment is an allogeneic hematopoietic stem cell transplant, better known as a bone marrow transplant (BMT). “This procedure replaces the child’s diseased blood system with healthy donor cells, offering the best chance for a cure,” explains the expert.

Benefits of bone marrow transplant

Dr Yadav shares how a bone marrow transplant works:

Conditioning: The child undergoes strong chemotherapy, sometimes with radiation, to destroy diseased bone marrow.

Transplant: Healthy blood-forming stem cells from a matched donor are infused via IV.

Engraftment: Donor cells settle in the bone marrow and start producing healthy blood cells.

Also read | Childhood cancer: Patterns of spread and early symptoms of Leukaemia, brain tumours, lymphomas

Understanding the risks

A compatible donor is critical, ideally a sibling with matching HLA markers. If unavailable, registries help find unrelated donors. Risks include:

• Graft-versus-host disease (donor cells attacking the child’s body)
• Serious infections due to weakened immunity
• Side effects from conditioning therapy
• Despite the risks, the potential to save a life makes the transplant essential.

Early care is crucial

JMML is rare, serious, and genetically driven, but early recognition and intervention with bone marrow transplantation can dramatically improve outcomes. “Research continues to advance, aiming to make treatments safer and more effective. There is hope, and timely care can truly change the course of this disease for children,” Dr Yadav emphasises.

FAQs on Juvenile Myelomonocytic Leukemia

1. What is Juvenile Myelomonocytic Leukemia (JMML)?

Juvenile Myelomonocytic Leukemia (JMML) is a rare and aggressive blood cancer affecting toddlers. It involves the overproduction of abnormal monocytes, a type of white blood cell, which can crowd out healthy cells and damage vital organs.

2. What causes JMML?

JMML is caused by mutations in specific genes such as PTPN11, NF1, CBL, NRAS, and KRAS. These mutations are usually acquired during a child's lifetime and lead to uncontrolled cell growth.

3. What are the symptoms of JMML?

Symptoms of JMML can resemble common childhood illnesses, including persistent fever, skin rashes, pale skin, easy bruising, unusual bleeding, frequent infections, and a swollen belly due to an enlarged liver and spleen.

4. How is JMML diagnosed?

JMML is diagnosed through blood tests that show high white cell counts, low red cells, and low platelets. Confirmation requires a bone marrow biopsy and genetic tests to identify specific mutations.

5. What is the most effective treatment for JMML?

The most effective treatment for JMML is an allogeneic hematopoietic stem cell transplant (bone marrow transplant). This procedure replaces the child's diseased blood system with healthy donor cells, offering the best chance for a cure.

Disclaimer: This article, including health and fitness advice, only provides generic information. Don’t treat it as a substitute for qualified medical opinion. Always consult a specialist for specific health diagnosis.