Profile of Pediatric Chronic Myeloid Leukemia in the Era of Imatinib - a Study from a Developing Country

INTRODUCTION

Chronic myeloid leukemia (CML) is very rare in children constituting 3% of pediatric leukemias and 10% of all CML cases. There is a lack of uniform guidelines, though the leukemic biology and clinicohematological features are the same as adults. Since the advent of imatinib, treatment modalities have evolved. However the effect of long term treatment on the child and family are areas to be studied, especially in developing countries.

AIM

To study the clinicohematologic profile, diagnostic and therapeutic methods, follow up measures, impact of the disease on the patient's education and the psychosocioeconomic compromise of the parents.

MATERIALS AND METHODS

Institutional ethics committee approval and informed consent from parents was obtained. All cases of pediatric CML were included. The patients and parents were interviewed and their medical records reviewed. The demography, clinical and hematological features at presentation, treatment, compliance, toxicity and follow up details were collected. The parents were provided with a validated questionnaire to assess the psychosocioeconomic impact.

RESULTS

In 2011 - 2014, 7 patients below 18 years of age were diagnosed to have CML. Incidence: 5.6 % of pediatric leukemias and 9 % of CML in our centre. 1 patient opted out of treatment; 6 are undergoing treatment and follow up. 3 (50%) were above 14 years of age, 2 (33%) between 10 and 14 years, 1 (17%) below 10 years. Male to female ratio is 2:1. The predominant presenting symptoms were abdominal pain, fever, weight loss and weakness. 4 patients (67%) complained of bone pain, pallor and throat pain. 4 patients (67%) had splenomegaly.

All patients presented at the chronic phase of CML. 50% had WBC count less than 20,000/µl and 1 patient (17%) had hyperleukocytosis. FISH and karyotyping detected t(9;22) in all patients. RT-PCR for bcr-abl was done. All patients were started on upfront therapy with standard dose of T.Imatinib. None needed escalated therapy. Toxic symptoms noted were gastrointestinal intolerance, skin rashes, hyperpigmentation, bone pain, myalgia and cramps which were tolerable in 5 (83%) patients. 1 (17%) had to be hospitalized. The dosage was reduced and then gradually increased. 5 patients (83%) were compliant.

RESPONSE TO TREATMENT AND FOLLOWUP

3 (50%) patients obtained full treatment response attaining complete molecular response (CMR) at 12-18 months, maintaining CMR at about 4 years of follow up.

2 (33%) patients are also responding well with major molecular response at 1 year (follow up 14 months) and complete cytogenetic response at 6 months (follow up 9 months). 1 patient showed treatment failure due to default follow up and loss of compliance.

PSYCHOSOCIOECONOMIC EFFECT:

4 (66%) patients absented from school for more than 6 months and lost an academic year.2 patients who were above 14 years of age absented for a few months which did not affect their studies.

5 (83%) patients belonged to upper middle socioeconomic class; they travelled to the hospital by bus and met medical expenses by insurance and support from funding agencies and friends. 1 (17%) belonged to upper class who met expenses by himself.

Of the parents of patients evaluated, 33% responded to feeling guilty. 16% felt guilt related to self, 33% felt guilt with regards to interpersonal relationships. 50% were depressed with 33% admitting to feeling gloomy, 33% feeling easily upset and 33% worried about the future. 50% showed difficulty in coping, 50% felt burdened, 50% felt their activities were affected. However, nobody turned to drugs or alcohol. 33% had problems in their interpersonal relationships; 17% had issues with family, 33% did not want to mingle with others. 17% felt stigmatized; 17% felt neglected, 17% felt their social life was disturbed. 33% felt economical constraints; 33% worried about making ends meet, 17% were required to sell/ mortgage assets.

DISCUSSION AND CONCLUSION:

In correlation with literature, pediatric CML is rare in our centre. Patients responded well with minimum toxicity to Imatinib when compliance was good. Schooling was affected by the disease. Parents showed signs of depression and difficulty in coping.

The government should initiate strategies to provide psychosocioeconomic support to CML patients and family members so they may cope with the disease.