Hematology is the specialty that learned to question its own best treatments.
In May 1995, the New England Journal of Medicine published a trial about sickle cell anemia. The disease bends red blood cells into a sickle shape, and the sickled cells cause attacks of severe pain. An earlier study had given patients a drug called hydroxyurea. It raised their fetal hemoglobin, and fetal hemoglobin can block the sickling. The trial asked whether hydroxyurea could reduce the pain attacks. It gave 299 adults with at least three attacks a year either hydroxyurea or a placebo. The trial was stopped early, after about 21 months. Patients on hydroxyurea had a median of 2.5 pain attacks a year. Patients on placebo had 4.5. Hydroxyurea also cut the need for blood transfusions, from 73 patients to 48. The drug caused no important side effects. An existing drug had nearly halved the pain attacks.
Almost seven years later, in January 2002, the same journal published a trial about lymphoma in older people. Diffuse large B-cell lymphoma is a fast-growing cancer of the white blood cells. The standard treatment was a mix of four chemotherapy drugs, called CHOP. Rituximab is an antibody that attaches to a marker on the lymphoma cells. The trial asked whether adding rituximab to CHOP would help. It gave 399 patients aged 60 to 80 either CHOP alone or CHOP with rituximab. The cancer disappeared completely in 76% of the patients given rituximab. It disappeared in 63% of the patients given CHOP alone. Rituximab also lowered the risk of death by about a third. It did not add serious side effects. An antibody aimed at the cancer cells had made the chemotherapy work better.
Eleven and a half years later, in July 2013, the same journal published a trial about acute promyelocytic leukemia. The standard treatment combined chemotherapy with a drug called ATRA. It cured more than 80% of patients. But the chemotherapy also damaged the blood, and early studies suggested that arsenic trioxide could replace it. The trial asked whether ATRA with arsenic, and no chemotherapy, could work as well. It treated 156 patients with either ATRA and arsenic or ATRA and chemotherapy. The arsenic group had 97% of patients alive without a relapse at two years. The chemotherapy group had 86%. Overall survival was also better with arsenic. Arsenic caused fewer infections and less harm to the blood, but more liver problems. A leukemia could now be treated without chemotherapy, and treated better.
These are three of the thousands of studies that shaped modern hematology. They taught hematology that an existing drug could nearly halve the pain attacks of sickle cell anemia, that an antibody aimed at lymphoma cells made chemotherapy work better without more harm, and that one leukemia could be treated better without chemotherapy at all, and each time the answer was in a paper years before it was in a textbook.
It is still happening. Recently, in September 2026, the New England Journal of Medicine published a trial about acute myeloid leukemia. Fit patients with this leukemia usually get intensive chemotherapy, which often causes severe side effects. Patients too frail for it get a gentler combination of two drugs, azacitidine and venetoclax. The trial asked whether fit patients might do better on the gentler combination too. It gave 172 patients either azacitidine and venetoclax or intensive chemotherapy. Most of them had high-risk disease. Patients on the gentler combination went a median of 14.5 months without a relapse, treatment failure or death. Patients on intensive chemotherapy went 6.2 months. Serious infections affected 28% of the patients on the combination. They affected 41% of the patients on chemotherapy. Serious bleeding fell from 12% to 2%. This was a phase 2 trial. The gentler treatment had beaten the harsh one. Practice follows the literature as it goes. It is a good culture. It has exactly one cost.
The literature grows faster than anyone can read it.
Across the medical topics a hematologist needs to stay up to date on, from leukemia and lymphoma to sickle cell disease and anticoagulation, around 390 full-text papers now arrive every week. That is about 1,700 a month, and more than 20,000 a year. Leukemia therapy alone is 75 of each week’s 390. Lymphoma treatment is 73. These are not press releases or opinion pieces. They are the same kind of paper as the sickle cell trial and the lymphoma trial, and somewhere in each week’s 390 are the handful that will change a prescription.
It is tempting to believe that experience closes the gap, that twenty years of clinic teach what the papers teach. The evidence points the other way. A systematic review of 62 studies found that in most of them, more years in practice went with lower adherence to current standards of care. Experience is not what keeps a hematologist current. Reading is. And reading is the one thing the week does not have room for.
So we built the tool you need to stay up to date.
We summarise the latest research for you and send it directly to your email inbox, every week, or whenever you choose.
A gentler azacitidine-venetoclax combination beat intensive chemotherapy in fit patients with AML
TakeawayIn 172 previously untreated adults with AML who were fit for induction chemotherapy, azacitidine plus venetoclax gave a median event-free survival of 14.5 months, against 6.2 with induction chemotherapy, with fewer serious infections and bleeds.
Induction chemotherapy has long been part of curative treatment for fit patients with AML, despite frequent severe side effects; hypomethylating therapy plus venetoclax is standard for those unfit for it. This phase 2 trial randomised 172 induction-eligible adults, median age 64, to azacitidine-venetoclax or induction chemotherapy.
- •Median event-free survival: 14.5 vs 6.2 months (hazard ratio 0.57, 95% CI 0.39 to 0.84; P = 0.002).