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 Preface

The pace of change in the field of pediatric hematology, oncology, and hematopoietic

cell therapies is staggering.

Molecular biol-ogy, genomics, and biochemistry have accel-erated the knowledge and understanding of disease states and further highlight the com-plex interplay of clinical, genetic, and social factors that constantly challenge us in therapid application of novel findings to treat patients with the goal of improved outcomes.

This translation of knowledge to the unique patient before us, the true art of the physi-

cian, encompassing experience, knowledge, intuition, and understanding of the individ-

ual needs and goals of patients and families, can be overwhelming.

What is needed is apractical, tested approach to analyze and address these problems to ensure timely evaluation, competent clinical care, and avoidance of pitfalls that might negatively impact the patient or future treatment options.

This practical approach is achieved by spending time with patients and families and observing the myriad variations in dis-ease and individual nuances that are not addressed in large studies or case reports, all the while expanding foundational knowledge.

This handbook represents the work of our colleagues at Children’s Hospital &

Research Center Oakland toward this endeavor.

The guidelines offered here have been used to instruct medical students,

pediatric residents, nurses, pediatricians, and hematology/oncology fellows for over

25 years.

This handbook provides clinical approaches for common problems in pedi-atric hematology, oncology, hematopoietic stem cell transplant, and newer cellular ther-apies; knowledge to organize and evaluate the care of your patients; and a framework to incorporate ever‐expanding psychosocial needs, clinical studies, medical treatments, and science.

All of these are essential com- ponents that encompass the care of the child with blood disorders and cancer.

Approach to the Anemic Child

Anemia is the condition in which the concentration of hemoglobin or the red

cell mass is reduced below normal.

Anemia results in a physiological decrease in the oxygen‐carrying capacity of the blood and

reduced oxygen supply to the tissues. Causes of anemia are increased loss or destruction

of red blood cells (RBCs) or a significant decreased rate of production.

When evalu- ating a child with anemia, it is important to determine if the problem is isolated to one cell line (e.g., RBCs) or multiple cell lines (i.e., RBCs, white blood cells [WBCs], or platelets). When two or three cell lines are affected, it may indicate bone marrow involvement (e.g., leukemia, metastatic dis- ease, and aplastic anemia), sequestration (i.e., hypersplenism), immune deficiency,

or an immune‐mediated process (e.g.,hemolytic anemia and immune thrombocy-

topenic purpura).

The evaluation of anemia includes a com- plete medical history, family history, physi-

cal examination, and laboratory assessment (see Figure 1.1).

The diagnosis of anemia is made after reference to established normal controls for

age (Table 1.1). The blood smear and red cell indices are very helpful in the diagnosis

and classification of anemia. They allow for classification by the cell size (mean corpus-

cular volume [MCV]), give the distribution of cell size (red cell distribution width [RDW]), and may give important diagnos- tic clues if specific morphological abnor- malities are present (e.g., sickle cells, target cells, and spherocytes). The MCV, RDW,

and reticulocyte count are helpful in the differential diagnosis of anemia.

A high RDW, or anisocytosis, is seen in stress erythropoiesis and is often suggestive of

iron deficiency or hemolysis. 

A normal or low reticulocyte count is an inappropriate response to anemia and suggests impaired red cell production. An elevated reticulo- cyte count suggests blood loss, hemolysis, or sequestration.